BioMarin to buy Amicus (NASDAQ: FOLD) in $14.50 cash deal
Filing Impact
Filing Sentiment
Form Type
10-K
Rhea-AI Filing Summary
Amicus Therapeutics files its annual report highlighting a pending all-cash acquisition by BioMarin and continued growth in rare-disease therapies. Under a December 2025 merger agreement, BioMarin will acquire Amicus for $14.50 per share in cash, after which Amicus will become a wholly owned subsidiary and its stock will cease trading publicly, contingent on shareholder and regulatory approvals.
Commercial performance is led by Fabry drug Galafold, which generated $521.7 million in 2025 revenue, up $63.6 million year over year, and Pompe therapy Pombiliti + Opfolda, with $112.5 million in 2025 revenue, an increase of $42.3 million. Cash, cash equivalents and marketable securities totaled $293.5 million as of December 31 2025, supporting ongoing commercialization and pipeline work, including the U.S. license for Phase 3 kidney candidate DMX‑200. The company also emphasizes extensive patent protection, global regulatory approvals, and a long list of risk factors spanning merger completion, competition, pricing, regulation, financing needs, and reliance on third parties.
Positive
- Definitive cash merger with BioMarin: Amicus agreed to be acquired under a merger whereby each share will be converted into the right to receive $14.50 in cash, subject to shareholder and regulatory approvals, providing a clear liquidity event for existing stockholders.
- Strong 2025 revenue growth in core franchises: Fabry therapy Galafold generated $521.7 million in revenue, up $63.6 million year over year, and Pompe treatment Pombiliti + Opfolda reached $112.5 million, up $42.3 million, showing commercial momentum in rare diseases.
Negative
- None.
Insights
Cash sale to BioMarin and rising rare‑disease revenues reshape Amicus’s outlook.
The centerpiece is a definitive merger agreement under which BioMarin will acquire Amicus for
Operationally, Amicus reports strong traction in commercial products. 2025 Galafold revenue reached
The company is also expanding via licensing, paying
UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM 10-K
(Mark One)
For the fiscal year ended December 31 , 2025
OR
For the transition period from to
Commission file number 001-33497
(Exact Name of Registrant as Specified in Its Charter)
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Securities registered pursuant to Section 12(b) of the Act:
| Title of each class | Trading Symbol(s) | Name of each exchange on which registered | ||||||
Securities registered pursuant to Section 12(g) of the Act: None
Indicate by check mark if the registrant is a well-known seasoned issuer, as defined in Rule 405 of the Securities Act. Yes ☒ No ☐
Indicate by check mark if the registrant is not required to file reports pursuant to Section 13 or Section 15(d) of the Act. Yes ☐ No ☒
Indicate by check mark whether the registrant (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange Act of 1934 during the preceding 12 months (or for such shorter period that the registrant was required to file such reports), and (2) has been subject to such filing requirements for the past 90 days. Yes ☒ No ☐
Indicate by check mark whether the registrant has submitted electronically every Interactive Data File required to be submitted pursuant to Rule 405 of Regulation S-T (§232.405 of this chapter) during the preceding 12 months (or for such shorter period that the registrant was required to submit such files). Yes ☒ No ☐
Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, a smaller reporting company, or an emerging growth company. See the definitions of "large accelerated filer," "accelerated filer," "smaller reporting company," and "emerging growth company" in Rule 12b-2 of the Exchange Act.
| ☒ | Accelerated filer | ☐ | ||||||||||||
| Non-accelerated filer | ☐ | Smaller reporting company | ||||||||||||
| Emerging growth company | ||||||||||||||
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐
Indicate by check mark whether the registrant has filed a report on and attestation to its management’s assessment of the effectiveness of its internal control over financial reporting under Section 404(b) of the Sarbanes-Oxley Act (15 U.S.C. 7262(b)) by the registered public accounting firm that prepared or issued its audit report .☒
If securities are registered pursuant to Section 12(b) of the Act, indicate by check mark whether the financial statements of the registrant included in the filing reflect the correction of an error to previously issued financial statements. ☐
Indicate by check mark whether any of those error corrections are restatements that required a recovery analysis of incentive-based compensation received by any of the registrant’s executive officers during the relevant recovery period pursuant to §240.10D-1(b). ☐
Indicate by check mark whether the registrant is a shell company (as defined in Rule 12b-2 of the Exchange Act). Yes ☐ No ☒
The aggregate market value of the 304,479,825 shares of voting common equity held by non-affiliates of the registrant, computed by reference to the closing price as reported on The NASDAQ Global Market, as of the last business day of the registrant's most recently completed second fiscal quarter (June 30, 2025) was $1,744,699,397 . Shares of voting and non-voting stock held by executive officers, directors, and holders of more than 10% of the outstanding stock have been excluded from this calculation because such persons or institutions may be deemed affiliates. This determination of affiliate status is not a conclusive determination for other purposes.
The number of shares outstanding of the registrant's common stock, $0.01 par value per share, as of February 12, 2026 was 314,000,712 shares.
Table of Contents
PART I | ||||||||
Item 1. | BUSINESS | 5 | ||||||
Item 1A. | RISK FACTORS | 26 | ||||||
Item 1B. | UNRESOLVED STAFF COMMENTS | 70 | ||||||
Item 1C. | CYBERSECURITY | 70 | ||||||
Item 2. | PROPERTIES | 72 | ||||||
Item 3. | LEGAL PROCEEDINGS | 72 | ||||||
Item 4. | MINE SAFETY DISCLOSURES | 72 | ||||||
PART II | ||||||||
Item 5. | MARKET FOR THE REGISTRANT'S COMMON EQUITY, RELATED STOCKHOLDER MATTERS AND ISSUER PURCHASES OF EQUITY SECURITIES | 73 | ||||||
Item 6. | [RESERVED] | 74 | ||||||
Item 7. | MANAGEMENT'S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS | 75 | ||||||
Item 7A. | QUANTITATIVE AND QUALITATIVE DISCLOSURES ABOUT MARKET RISK | 81 | ||||||
Item 8. | FINANCIAL STATEMENTS AND SUPPLEMENTARY DATA | 83 | ||||||
Item 9. | CHANGES IN AND DISAGREEMENTS WITH ACCOUNTANTS ON ACCOUNTING AND FINANCIAL DISCLOSURE | 120 | ||||||
Item 9A. | CONTROLS AND PROCEDURES | 120 | ||||||
Item 9B. | OTHER INFORMATION | 120 | ||||||
Item 9C. | DISCLOSURE REGARDING FOREIGN JURISDICTIONS THAT PREVENT INSPECTIONS | 120 | ||||||
PART III | ||||||||
Item 10. | DIRECTORS, EXECUTIVE OFFICERS, AND CORPORATE GOVERNANCE | 121 | ||||||
Item 11. | EXECUTIVE COMPENSATION | 129 | ||||||
Item 12. | SECURITY OWNERSHIP OF CERTAIN BENEFICIAL OWNERS AND MANAGEMENT AND RELATED STOCKHOLDER MATTERS | 162 | ||||||
Item 13. | CERTAIN RELATIONSHIPS AND RELATED TRANSACTIONS AND DIRECTOR INDEPENDENCE | 165 | ||||||
Item 14. | PRINCIPAL ACCOUNTING FEES AND SERVICES | 165 | ||||||
PART IV | ||||||||
Item 15. | EXHIBITS, FINANCIAL STATEMENT SCHEDULES | 168 | ||||||
Item 16. | FORM 10-K SUMMARY | 173 | ||||||
SIGNATURES | 174 | |||||||
We have filed applications to register certain trademarks in the United States and abroad, including AMICUS THERAPEUTICS and design, Galafold® and design, Pombiliti® and design, Opfolda® and design.
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SPECIAL NOTE REGARDING FORWARD-LOOKING STATEMENTS
This Annual Report on Form 10-K contains forward-looking statements that involve risks, uncertainties, and assumptions. Forward-looking statements are all statements, other than statements of historical facts, that discuss our current expectation and projections relating to our strategy, future operations, future financial position, future revenues, projected costs, prospects, plans, and objectives of management. These statements may be preceded by, followed by or include the words "aim," "anticipate," "believe," "can," "could," "estimate," "expect," "forecast," "intend," "likely," "may," "might," "outlook," "plan," "potential," "predict," "project," "seek," "should," "will," "would," the negatives or plurals thereof, and other words and terms of similar meaning, although not all forward-looking statements contain these identifying words.
We have based these forward-looking statements on our current expectations and projections about future events. Although we believe that our assumptions made in connection with the forward-looking statements are reasonable, we cannot assure you that the assumptions and expectations will prove to be correct. You should understand that the following important factors could affect our future results and could cause those results or other outcomes to differ materially from those expressed or implied in our forward-looking statements:
•the occurrence of any event, change or other circumstance that could give rise to the termination of the definitive merger agreement (the "Merger Agreement") that we entered into with BioMarin Pharmaceutical Inc. ("BioMarin"), and its wholly owned acquisition subsidiary on December 19, 2025, pursuant to which we expect to become a wholly owned subsidiary of BioMarin;
•the failure to satisfy required closing conditions under the Merger Agreement, including, but not limited to, adoption of the Merger Agreement by the holders of at least a majority of the outstanding shares of our common stock and the receipt of required regulatory approvals, or the failure to complete the transaction in a timely manner;
•risks related to disruption of management's attention from our ongoing business operations due to the pendency of the transaction with BioMarin;
•the effect of the announcement of the transaction with BioMarin on our operating results and business generally, including, but not limited to, our ability to retain and hire key personnel and maintain our relationships with business partners, manufacturers, customers, vendors, regulatory authorities and others with whom we do business;
•the impact of the pending transaction with BioMarin on our strategic plans and operations and our ability to respond effectively to competitive pressures, industry developments and future opportunities;
•the scope, progress, results and costs of clinical trials for our drug candidates;
•the cost of manufacturing drug supply for our commercial, clinical and preclinical studies, including the cost of manufacturing Pombiliti® (also referred to as "ATB200" or "cipaglucosidase alfa");
•the future results of preclinical research and subsequent clinical trials for pipeline candidates we may identify from time to time, including our ability to obtain regulatory approvals and commercialize such therapies;
•the costs, timing, and outcome of regulatory review of our product candidates;
•any changes in regulatory standards relating to the review of our product candidates;
•any changes in laws, rules or regulations, including the imposition of tariffs, most favored nation requirements, or other trade restrictions or requirements, affecting our ability to manufacture, transport, test, develop, or commercialize our products, including Galafold®, Pombiliti® + Opfolda®, or our product candidates;
•the costs of commercialization activities, including product marketing, sales, and distribution;
•the emergence of competing technologies and other adverse market developments;
•the estimates regarding the potential market opportunity for our products and product candidates;
•our ability to successfully commercialize Galafold® (also referred to as "migalastat HCl");
•our ability to successfully commercialize Pombiliti® + Opfolda® in the E.U., U.K., and U.S., and elsewhere, if regulatory applications are approved;
•our ability to manufacture or supply sufficient clinical or commercial products, including Galafold® and Pombiliti® + Opfolda®;
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•our ability to obtain reimbursement for Galafold® and Pombiliti® + Opfolda®;
•our ability to satisfy post-marketing commitments or requirements for continued regulatory approval of Galafold® and Pombiliti® + Opfolda®;
•our ability to obtain market acceptance of Galafold® and Pombiliti® + Opfolda®, or any other product developed or acquired that has received regulatory approval;
•the costs of preparing, filing, and prosecuting patent applications and maintaining, enforcing, and defending intellectual property-related claims, including Hatch-Waxman litigation;
•the impact of litigation that has been or may be brought against us or of litigation that we are pursuing or may pursue against others, including Hatch-Waxman litigation;
•the extent to which we acquire or invest in businesses, products, and technologies;
•our ability to successfully integrate acquired products and technologies into our business, or successfully divest or license existing products and technologies from our business, including the possibility that the expected benefits of the transactions will not be fully realized by us or may take longer to realize than expected;
•our ability to establish licensing agreements, collaborations, partnerships or other similar arrangements and to obtain milestone, royalty, or other economic benefits from any such collaborators;
•the costs associated with, and our ability to comply with, emerging sustainability standards, including climate reporting requirements at the local, state and national levels, especially abroad;
•our ability to successfully protect our information technology systems and maintain our global operations and supply chain without interruption;
•our ability to accurately forecast revenue, operating expenditures, or other metrics impacting profitability;
•fluctuations in foreign currency exchange rates; and
•changes in accounting standards.
In light of these risks and uncertainties, we may not actually achieve the plans, intentions or expectations disclosed in our forward-looking statements, and you should not place undue reliance on our forward-looking statements. Actual results or events could differ materially from the plans, intentions, and expectations disclosed in the forward-looking statements we make. We have included important factors in the cautionary statements included in this Annual Report on Form 10-K, particularly in Part I, Item 1A "— Risk Factors", a summary of which may be found below, that we believe could cause actual results or events to differ materially from the forward-looking statements that we make. Those factors and the other risk factors described herein are not necessarily all of the important factors that could cause actual results or developments to differ materially from those expressed in any of our forward-looking statements. Other unknown or unpredictable factors also could harm our results. Our forward-looking statements do not reflect the potential impact of any future collaborations, alliances, business combinations, partnerships, strategic out-licensing of certain assets, the acquisition of preclinical-stage, clinical-stage, marketed products or platform technologies or other investments we may make and do not assume the consummation of our pending transaction with BioMarin unless specifically stated otherwise. Consequently, there can be no assurance that actual results or developments anticipated by us will be realized or, even if substantially realized, that they will have the expected consequences to, or effects on, us. Given these uncertainties, investors are cautioned not to place undue reliance on such forward-looking statements.
You should read this Annual Report on Form 10-K and the documents that we incorporate by reference in this Annual Report on Form 10-K completely and with the understanding that our actual future results may be materially different from what we expect. These forward-looking statements speak only as of the date of this report. We undertake no obligation, and specifically decline any obligation, to publicly update or revise any forward-looking statements, even if experience or future developments make it clear that projected results expressed or implied in such statements will not be realized, except as may be required by law.
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Summary Risk Factors
The following is a summary of the principal risks that make an investment in our common stock speculative or risky. This summary does not address all of the risks that we face and is qualified in its entirety by reference to the more detailed descriptions included in Part I, Item 1A "— Risk Factors". This summary should be read together with those more detailed descriptions, along with our other SEC filings before making an investment decision.
•We may not complete the pending transaction with BioMarin within the time frame we anticipate or at all, which could have an adverse effect on our business, financial results and/or operations.
•While the Merger Agreement is in effect, we are subject to restrictions on our business activities.
•We depend heavily on sales of Galafold® and, increasingly, Pombiliti® + Opfolda®. If we are delayed or unable to commercialize these products successfully, our business could be materially harmed.
•If we are not able to obtain, or delayed in obtaining, required regulatory approvals, we will not be able to commercialize our products or product candidates, materially impairing our ability to generate revenue.
•If we are unable to establish and maintain sales and marketing capabilities, or relationships, for our products or, if approved, product candidates, their commercialization may suffer.
•If the market opportunities for our products or product candidates are smaller than we believe they are, then our revenues may be adversely affected, and our business may suffer.
•Galafold®, Pombiliti® + Opfolda®, or any of our product candidates that receive regulatory approval may fail to achieve the degree of market acceptance necessary for commercial success.
•We face substantial competition, which may result in others discovering, developing or commercializing products before or more successfully than we do.
•A variety of risks associated with international operations, including the U.S. and China relations, could adversely affect our business, particularly as it relates to products or product candidates for which we have a primary supplier.
•Our products or any product candidates receiving approval may become subject to unfavorable pricing regulations, third-party coverage and reimbursement practices or healthcare reform initiatives.
•If we are found to have promoted off-label uses by regulatory authorities, we may become subject to significant liability.
•Product liability lawsuits against us could cause us to incur substantial liabilities and limit commercialization of any products that we may develop.
•If applicable regulatory authorities approve generic or biosimilar products with claims that compete with our products or any of our product candidates, it could reduce our sales.
•We may expend our limited resources to pursue a particular product, product candidate or indication and fail to capitalize on an alternative for which there is a greater likelihood of success.
•Our products or product candidates may have side effects that could impact their regulatory approval or commercialization.
•Any product or product candidate we obtain marketing approval for could be subject to restrictions or withdrawal from the market and we may be subject to penalties or enforcement actions if we fail to comply with regulatory requirements.
•Certain relationships will be subject to anti-kickback, fraud and abuse, anti-bribery and corruption and other laws and regulations, which could expose us to criminal, civil, or contractual penalties, reputational harm and diminished earnings.
•If clinical trials of our product candidates, including DMX-200, do not produce results satisfactory to regulatory authorities, the development and commercialization of our product candidates may not be completed.
•If we experience delays or difficulties in the enrollment of, or other unforeseen events in connection with, our clinical trials, potential regulatory approval or commercialization of our product candidates could be delayed or prevented.
•If our competitors obtain orphan drug exclusivity for their products and we do not, we may be unable to have competing products approved in the applicable jurisdiction for a significant period of time.
•Failure to obtain or maintain regulatory approval outside the U.S. would prevent us from marketing our products abroad.
•Our use of third parties to manufacture our products or product candidates may increase the risk that we will not have sufficient quantities of our products or product candidates or such quantities at an acceptable cost, which could delay, prevent or impair our development or commercialization efforts.
•We may be unable to enter into agreements with third-party manufacturers, or unable to do so on acceptable terms.
•We rely on third parties to distribute our products who may not perform satisfactorily.
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•We rely on third parties to conduct certain preclinical activities and our clinical trials, who may not perform satisfactorily.
•We may not be successful in maintaining or establishing collaborations, which could adversely affect our ability to develop and, particularly in international markets, commercialize products.
•Materials necessary to manufacture our products or product candidates may not be available on commercially reasonable terms, which may delay their development and commercialization.
•Manufacturing issues may arise that could increase costs or delay commercialization.
•We have incurred significant losses and anticipate that we will continue to incur losses in the future.
•We may never become profitable even though we currently generate revenue from the sale of products.
•If we require, and fail to obtain, additional necessary financing, we may be unable to complete the development and commercialization of our products and product candidates.
•Raising additional capital may cause dilution to our existing stockholders, restrict our operations, or require us to relinquish rights to our technologies, Galafold®, Pombiliti® + Opfolda®, or product candidates.
•We may not have sufficient cash flow from our business to pay our substantial debt.
•Foreign currency exchange rate fluctuations could harm our financial results.
•Our ability to use our net operating loss carryforwards and certain other tax attributes may be limited.
•Our executive officers, directors and principal stockholders maintain the ability to exert significant influence and control over matters submitted to our stockholders for approval.
•We do not anticipate paying cash dividends so capital appreciation, if any, will be our stockholders sole source of gain.
•Our disclosure controls and procedures may not prevent or detect all errors or acts of fraud.
•If we are unable to obtain and maintain sufficiently broad patent protection, our ability to successfully commercialize our technology and products may be adversely affected.
•We currently are and may become involved in lawsuits to protect or enforce our patents or other intellectual property.
•Third parties may initiate legal proceedings alleging that we are infringing their intellectual property rights which could have a material adverse effect on the success of our business.
•We may be subject to claims by third parties asserting that we or our employees have misappropriated their intellectual property, or claiming ownership of what we regard as our own intellectual property.
•If we fail to comply with our obligations in our intellectual property licenses, we could lose material license rights.
•Failure to secure trademark registrations could adversely affect our business.
•Our future success depends on our ability to retain key executives and to attract, retain and motivate qualified personnel.
•We expect to expand our development, regulatory and sales and marketing capabilities, and as a result, we may encounter difficulties in managing our growth, which could disrupt our operations.
•Our employees, independent contractors, principal investigators, consultants and vendors may engage in misconduct or improper activities which could lead to significant liability and harm our reputation.
•If our enterprise risk program, global risk committee and other compliance methods are not effective, our business, financial condition and operating results may be adversely affected.
•The increased focus on sustainability reporting by investors, governmental bodies and other stakeholders, as well as existing and proposed laws related to this topic, may adversely affect our business and reputation.
•Our business may be impacted by actions of the current U.S. government, including executive orders, policies, new legislation, and judicial decisions.
•Our business activities involve the use of hazardous materials which could subject us to significant adverse consequences if we fail to comply with the applicable laws regulating their use.
•Unfavorable global economic conditions such as global crises, health epidemics, military conflicts, geopolitical and trade disputes, including between the U.S. and China, or other factors, may adversely affect our business and financial results.
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PART I
Item 1. BUSINESS
Overview
We are a leading, global biotechnology company with a clear and compelling mission to develop and deliver transformative medicines for people living with rare diseases. With extraordinary patient focus, we strive to redefine expectations in rare disease. Our two marketed therapies are Galafold®, the first oral monotherapy for people living with Fabry disease who have amenable genetic variants, and Pombiliti® + Opfolda®, a novel two-component treatment for adults living with late-onset Pompe disease.
Galafold® (also referred to as "migalastat") is approved in over 40 countries around the world, including the United States ("U.S."), European Union ("E.U."), United Kingdom ("U.K."), and Japan. Additionally, Galafold® has been granted orphan drug designation in the U.S., E.U., U.K., Japan, and several other countries.
Pombiliti® + Opfolda® (also referred to as "cipaglucosidase alfa-atga/miglustat") is approved in the U.S., the E.U., the U.K., Canada, Australia, Switzerland, and Japan. Multiple regulatory submissions and reimbursement processes with global health authorities are currently underway. Additionally, Pombiliti® + Opfolda® has been granted orphan drug designation or status in the U.S., U.K., Switzerland and Japan and data exclusivity in the E.U.
On April 30, 2025, we entered into an exclusive license agreement with Dimerix Bioscience Pty Limited ("Dimerix") for the U.S. commercialization rights of Dimerix' Phase 3 drug candidate, DMX-200 for treatment of Focal Segmental Glomerulosclerosis ("FSGS") and other indications.
On December 19, 2025, we entered into a definitive merger agreement (the "Merger Agreement") with BioMarin Pharmaceutical Inc. ("BioMarin") and its wholly owned acquisition subsidiary. Under the terms of the Merger Agreement, and in accordance with the General Corporation Law of the State of Delaware (the "DGCL"), if the transaction is completed, BioMarin's wholly owned acquisition subsidiary will merge with and into Amicus (the "Merger"), and Amicus will continue as the surviving corporation as a wholly owned subsidiary of BioMarin. In addition, all shares of our common stock outstanding, immediately prior to closing (other than Excluded Shares or Dissenting Shares (as defined in the Merger Agreement)) will be cancelled and converted into the right to receive $14.50 per share in cash, without interest and subject to any applicable withholding of taxes (the "Merger Consideration"). Following completion of the Merger, shares of our common stock will no longer be publicly listed. The transaction is expected to close in the second quarter of 2026, subject to regulatory clearances, approval by our stockholders and other customary closing conditions. Refer to "— Note 15. Merger Agreement," in our Notes to Consolidated Financial Statements for additional information.
Our Strategy
Our strategy is to create, manufacture, test, and deliver the highest quality medicines for people living with rare diseases through internally developed, jointly developed, acquired, or in-licensed products and product candidates. We are leveraging our global capabilities to develop and broaden our franchises in Fabry and Pompe disease, with focused discovery work on next-generation therapies and novel technologies.
Highlights of our progress include:
•Commercial success in Fabry disease. For the year ended December 31, 2025, Galafold® revenue was $521.7 million of consolidated revenue, which represented an increase of $63.6 million compared to the prior year. We continue to see strong commercial momentum and expansion into additional geographies.
•Commercial and regulatory success in Pompe disease. For the year ended December 31, 2025, Pombiliti® + Opfolda® revenue was $112.5 million of consolidated revenue, which represented an increase of $42.3 million compared to the prior year. Pombiliti® + Opfolda® has been approved by the respective regulatory authorities in the E.U., U.S., U.K., Canada, Switzerland, Australia, and Japan. Additionally, throughout 2024 and 2025, we established reimbursement agreements in multiple countries.
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•Pipeline advancement and growth. On April 30, 2025, Amicus licensed exclusive rights to commercialize DMX-200, a small molecule currently in a pivotal Phase 3 study ("ACTION3"), in the U.S. for treatment of FSGS and other indications.
•Financial strength. Total cash, cash equivalents, and marketable securities as of December 31, 2025 was $293.5 million.
Our Commercial Products and Product Candidates
Galafold® (migalastat HCl) for Fabry Disease
Our oral precision medicine Galafold® was granted accelerated approval by the FDA in August 2018 for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene ("GLA") variant based on in vitro assay data. Galafold® was approved in the E.U. and U.K. in May 2016 as a first-line therapy for long-term treatment of adults and adolescents, aged 16 years and older, with a confirmed diagnosis of Fabry disease and who have an amenable variant. Marketing authorization approvals as well as approvals for adolescents aged 12 years and older weighing 45 kg or more have been granted in over 40 countries around the world. We plan to continue to launch Galafold® in additional countries, upon receipt of marketing authorization.
As an orally administered monotherapy, Galafold® is designed to bind to and stabilize an endogenous alpha-galactosidase A ("alpha-Gal A") enzyme in those patients with genetic variants identified as amenable in a Good Laboratory Practice ("GLP") cell-based amenability assay.
Fabry Disease Background
Patients with Fabry disease have an inherited deficiency of the alpha-Gal A enzyme that would normally degrade the lipid substrate globotriaosylceramide in the lysosome. Genetic variants that cause changes in the amino acid sequence of alpha-Gal A result in an unstable enzyme that does not efficiently fold into its correct three-dimensional shape and cannot be trafficked properly in the cell, even if it has the potential for biological activity. Galafold® is an oral small molecule pharmacological chaperone that is designed to bind to and stabilize a patient’s own endogenous target protein. This is considered a precision medicine because Galafold® targets only patients with GLA variants amenable to Galafold®.
Fabry disease is an X-linked disease caused by mutations in the GLA gene, which encodes the alpha-Gal A enzyme. These mutations can cause alpha-Gal A to be either absent or deficient. When alpha-Gal A is absent or deficient in the substrates, GL-3 and lyso-Gb3 accumulate, leading to damage of cells within affected parts of the individual's body and causing the various pathologies seen in Fabry disease. Fabry disease leads to progressive, irreversible organ damage, typically involving the nervous, cardiac, and renal systems, as well as multiple other tissues. The symptoms can be severe, differ from patient to patient, and begin at an early age, resulting in significant clinical, humanistic, and healthcare costs. Fabry disease requires lifelong medical intervention to manage the complications of this devastating disease across multiple organ systems.
Fabry disease is a relatively rare disorder. The annual incidence of Fabry disease in newborn males has been historically estimated to be 1:40,000-1:60,000 (Journal of the American Medical Association January 1999 and The Metabolic and Molecular Bases of Inherited Disease 8th edition 2001). However, more recent newborn screening studies in Italy, Taiwan, Austria, Spain and the U.S., which collectively screened more than 500,000 male and female newborns, found the incidence of GLA mutations to be between 1:2,445 to 1:8,454, more than ten times higher than previous estimates for classic patients (American Journal of Human Genetics 2006, Human Mutation 2009, the Lancet 2011, Journal of Pediatrics 2017, and Journal of the American Medical Association Pediatrics 2018). When looking at only male newborns within these studies, the incidence of Fabry disease mutations is as high as 1:1,316 – 1:7,575 (Circulation in Cardiovascular Genetics 2009, American Journal of Human Genetics 2006, European Journal of Pediatrics 2017).
We believe that approximately 35-50% of the Fabry disease patient population may benefit from treatment with Galafold® as a monotherapy. Additionally, we expect that as awareness of late-onset symptoms of Fabry disease grows, the number of patients diagnosed with the disease will increase. Increased awareness of Fabry disease, particularly for specialists not accustomed to treating Fabry disease patients, may lead to increased testing and diagnosis of patients with the disease. A number of factors are contributing to the continued expansion of Fabry diagnosis, including newborn screening in several states in the U.S., family screening, and access to lower cost genetic testing.
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Currently, three other products, all ERTs, are approved for the treatment of Fabry disease: agalsidase beta by Sanofi Aventis, pegunigalsidase alfa-iwxj by Chiesi Farmaceutici and agalsidase alfa by Takeda, the last of which is not approved in the U.S.
Pombiliti® (cipaglucosidase alfa-atga) + Opfolda® (miglustat) for Pompe Disease
We have leveraged our biologics capabilities to develop Pombiliti® + Opfolda®, a novel two-component treatment paradigm for Pompe disease. Pombiliti® + Opfolda® was approved by the European Commission ("EC"), the Medicines and Healthcare products Regulatory Agency ("MHRA"), and the Food and Drug Administration ("FDA") in 2023, the Swissmedic and the Therapeutics Good Administration in Australia in 2024, and Health Canada and Japan's Ministry of Health, Labor and Welfare in 2025 for adult late-onset Pompe disease ("LOPD") patients. Additional regulatory submissions and reimbursement processes with global health authorities are currently underway.
Pombiliti® + Opfolda® consists of a uniquely engineered recombinant human acid alpha-glucosidase ("rhGAA") enzyme, cipaglucosidase alfa-atga, with an optimized carbohydrate structure to enhance cellular uptake, administered intravenously in combination with orally administered miglustat. Miglustat binds to and stabilizes the cipaglucosidase alfa-atga in circulation reducing inactivation of rhGAA in circulation to improve the uptake of active enzyme into key disease relevant tissues. Miglustat is not an active ingredient that contributes directly to glycogen reduction.
In addition, clinical studies are ongoing in pediatric patients for both the LOPD and infantile-onset Pompe disease ("IOPD") populations.
Pompe Disease Background
Pompe disease is a lysosomal storage disorder that results from a deficiency in an enzyme, GAA. Signs and symptoms of Pompe disease can be severe and debilitating and include progressive muscle weakness throughout the body, particularly the heart and skeletal muscles. GAA deficiency causes accumulation of glycogen in cells, which is believed to result in the clinical manifestations of Pompe disease. Pompe disease ranges from a rapidly fatal infantile form with severe cardiac involvement to a more slowly progressive, late-onset form primarily affecting skeletal muscle. All forms are characterized by severe muscle weakness that worsens over time. In the early-onset form, patients are usually diagnosed shortly after birth and often experience enlargement of the heart and severe muscle weakness. In late-onset Pompe disease, symptoms may not appear until late childhood or adulthood and patients often experience progressive muscle weakness.
According to reported estimates of the Acid Maltase Deficiency Association, the United Pompe Foundation, and the Lysosomal Disease Program at Massachusetts General Hospital, there are 5,000-10,000 patients with Pompe disease worldwide. Pompe disease is a rare genetic disease with a traditionally used incidence rate of 1:40,000 (European Journal of Human Genetics 1999). However, it’s increasingly recognized that the incidence rate varies among different ethnic groups, forms of the disease (infantile onset vs late onset Pompe Disease) and countries (Molecular Genetics and Metabolism Reports 2021). With the advent of new-born screening and adoption by several states in the U.S. and elsewhere, more definitive incidence rate data is beginning to be gathered with rates as low as 1:10,152 (Current Treatment Options Neurology 2022). Based on a recent study of population genetic prevalence, the revised estimated incidence rate is now believed to be 1:23,232 (Molecular Genetics and Metabolism Reports 2021). As additional newborn screening data is gathered worldwide, a clearer picture of the disease epidemiology will emerge.
Currently, two products, both ERTs, are approved for the treatment of Pompe disease: alglucosidase alfa and avalglucosidase alfa-ngpt by Sanofi Aventis.
DMX-200 for Focal Segmental Glomerulosclerosis (FSGS)
DMX-200 is a small molecule inhibitor of the chemokine receptor 2 (CCR2) under development in a pivotal Phase 3 study (ACTION3), for the treatment of FSGS kidney disease. In early 2024, Dimerix reported positive interim results from the ACTION3 trial in FSGS showing DMX-200 was performing better than placebo in reducing proteinuria with no safety concerns to date. An additional blinded interim analysis is planned once the revised primary and secondary endpoints have been pre-specified in the protocol and agreed with the FDA. In a March 2025 Type C meeting, Dimerix successfully aligned with the FDA on proteinuria as an appropriate primary endpoint for traditional marketing approval for DMX-200. In December of 2025, Dimerix announced completion of enrollment for ACTION3.
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FSGS is a rare, serious kidney disorder characterized by progressive scarring (sclerosis) in parts of the glomeruli—the kidney’s filtering units. This scarring leads to leakage of protein into the urine (proteinuria), and progressive loss of kidney function leading to end-stage kidney disease. FSGS is increasingly understood to have an inflammatory component, with monocyte and macrophage activation contributing to glomerular injury. In the United States, more than 40,000 people are estimated to be living with FSGS, including both adults and children. There are no therapies specifically approved for FSGS in the U.S. Management of the disease relies on non-specific immunosuppressive and supportive therapies. In patients with progressive or treatment-resistant FSGS, the average time from diagnosis to end-stage kidney disease can be as short as five years. Even among those who undergo kidney transplantation, disease recurrence occurs in up to 60% of cases, underscoring the urgent need for new, disease-modifying treatments. DMX-200 is the only clinical-stage kidney disease program that specifically targets monocyte-driven inflammation, a mechanistic approach distinct from all other known therapies in development. Several other agents are in late-stage clinical development for FSGS, targeting alternative pathways including endothelin receptor antagonists and APOL1 inhibitors. Preclinical in vitro data supports potential mechanistic synergy between DMX-200 and endothelin antagonists.
Strategic Alliances and Arrangements
We will continue to evaluate business development opportunities to build stockholder value and provide us with access to the financial, technical, clinical, commercial resources, and intellectual property necessary to develop and market technologies or products in rare and orphan diseases. We are exploring potential collaborations, alliances, and various other business development opportunities on a regular basis. These opportunities may include business combinations, partnerships, the strategic in-licensing or out-licensing of certain assets, or the acquisition of preclinical-stage, clinical-stage, or marketed products or novel technologies consistent with our corporate strategy to develop and provide therapies to patients living with rare and orphan diseases.
Intellectual Property
Patents and Trade Secrets
Our success depends in part on our ability to maintain proprietary protection surrounding our product candidates, technology, and know-how, to operate without infringing the proprietary rights of others, and to prevent others from infringing our proprietary rights. Our policy is to seek to protect our proprietary position by filing U.S. and foreign patent applications related to our proprietary technology, including both new inventions and improvements of existing technology, that are important to the development of our business, unless this proprietary position would be better protected using trade secrets. Our patent strategy includes obtaining patent protection, where possible, on compositions of matter, methods of manufacture, methods of use, combination therapies, dosing and administration regimens, formulations, therapeutic monitoring, screening methods, and assays. We also rely on trade secrets, know-how, continuing technological innovation, in-licensing, and partnership opportunities to develop and maintain our proprietary position. Lastly, we monitor third parties for activities that may infringe our proprietary rights, as well as the progression of third-party patent applications that may have the potential to create blocks to our products or otherwise interfere with the development of our business. If any of these patents were to be asserted against us, there is no assurance that a court would find in our favor or that, if we choose or are required to seek a license, a license to any of these patents would be available to us on acceptable terms or at all.
We own or hold license rights to several issued patents and numerous pending and issued applications, filed in the U.S., Europe, Japan, and other jurisdictions that are related to Galafold® and our ongoing clinical programs:
•We own issued U.S. patents that cover the use of migalastat, the active pharmaceutical ingredient in Galafold®, in the treatment of Fabry disease, which expire between 2027 and 2042 and are listed in the FDA Orange Book. Foreign counterparts of the U.S. patents are pending or issued in Europe, Japan, and certain other jurisdictions. Further, we have pending U.S. patent applications, as well as their foreign counterparts covering various aspects of Galafold®, including composition-of-matter, methods of treating a patient diagnosed with Fabry disease, and methods of manufacturing. Some of the applications have been issued as patents, with the latest one expiring in 2043. We anticipate listing these patents in the FDA Orange Book if issued.
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•We own several issued U.S. patents that cover various aspects of Opfolda® and Pombiliti®, a pharmacological chaperon/ERT combination in the treatment of Pompe disease, which expire between 2033 and 2038. Several of the issued U.S. patents are listed in the FDA Orange Book for Opfolda®. Foreign counterparts to the issued patents are pending or issued in Europe, Japan, and certain other jurisdictions. We also have pending U.S. patent applications, as well as foreign counterpart applications, covering various aspects of compositions, methods of treatment, methods of manufacture, and formulations. Any patents issuing from these pending applications will expire between 2033 and 2043. We also filed Patent Term Extension application at the U.S. Patent and Trademark Office ("USPTO"), requesting that the term of certain issued U.S. patent covering cipaglucosidase alfa, the active pharmaceutical ingredient in Pombiliti®, be extended pursuant to 35 U.S.C. § 156.
Patent term extensions and adjustments, supplementary protection certificates, and pediatric exclusivity periods are not reflected in the expiration dates listed above and may extend protection.
In addition to our clinical programs, we actively monitor and file patent applications in the U.S. and in foreign countries on relevant technologies and pre-clinical programs. For example, we own or hold license rights to U.S. and foreign patents or patent applications covering the following:
•Next-generation Fabry chaperones;
•Gene therapy protein engineering technology;
•Gene therapy (e.g., Pompe, Fabry) programs and their use to treat specified diseases. We cannot be certain, however, that issued patents will be enforceable or provide adequate protection or that pending patent applications will result in issued patents.
•Individual patents extend for varying periods depending on the effective date of filing of the patent application or the date of patent issuance, and the legal term of the patents in the countries in which they are obtained. Generally, patents issued in the U.S. are effective for 20 years from the earliest nonprovisional filing date. This period may be shortened by terminal disclaimer or further extended by patent term adjustment or extension. The term of foreign patents varies in accordance with provisions of applicable local law, but typically is 20 years from the earliest nonprovisional filing date.
The U.S. Drug Price Competition and Patent Term Restoration Act of 1984, and amendments thereto, more commonly known as the Hatch-Waxman Act, provides for an extension of one patent, known as a Hatch-Waxman statutory extension, for each New Chemical Entity ("NCE") to compensate for a portion of the time spent in clinical development and regulatory review. However, the maximum extension is five years and the extension cannot extend the patent beyond 14 years from the New Drug Application ("NDA") approval. Similar extensions are available in European countries, known as Supplemental Protection Certificate ("SPC") extensions, Japan, and other countries. In addition, in the U.S., under provisions of the Best Pharmaceuticals for Children Act, we may be entitled to an additional six-month period of patent protection or market exclusivity for completing pediatric clinical studies in response to an FDA issued Pediatric Written Request before said exclusivities expire.
In the fourth quarter of 2022, we received Paragraph IV Certification Notice Letters from Teva Pharmaceuticals Inc. ("Teva"), Aurobindo Pharma Limited ("Aurobindo"), and Lupin Ltd. ("Lupin") in connection with Abbreviated New Drug Applications (“ANDA”) filed with the FDA requesting approval to market generic Galafold®. We filed lawsuits against Teva, Lupin, and Aurobindo in the U.S. District Court for the District of Delaware (the "District Court") for infringement of our Orange Book-listed patents. Lupin, Aurobindo, and Teva supplemented their Paragraph IV Certifications in 2023. In the fourth quarter of 2023, a stipulation order to stay litigation with respect to Lupin was ordered. Additionally, in the first quarter of 2024, a stipulation was filed with the court and approved by the presiding judge, whereby the parties agreed to accept our definition of a term that was in dispute. As such, the scheduled Markman hearing was deemed unneeded and cancelled.
In October 2024, we entered into a non-exclusive, non-transferable, royalty-free license with Teva which will allow Teva to market its generic version of Galafold® in the United States beginning on January 30, 2037, or earlier in certain circumstances. In accordance with the license agreement, a consent judgment and permanent injunction was entered with the District Court and all Hatch-Waxman litigation between Amicus and Teva has been terminated. As required by law, Amicus and Teva have submitted the confidential license agreement to the U.S. Federal Trade Commission and the U.S. Department of Justice for review.
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From September 29, 2025 to October 1, 2025, the District Court held a three-day trial to hear evidence and argument as to the disputed patent issues between Aurobindo and Amicus. On December 19, 2025, Amicus announced that it had resolved the patent litigation with Aurobindo and Lupin. In connection with the resolution of the patent litigation, Amicus entered into non-exclusive, non-transferable, royalty-free licenses with Aurobindo and Lupin respectively, which will allow Aurobindo and Lupin to market their respective generic versions of Galafold® in the U.S. beginning on January 30, 2037, or earlier in certain circumstances. As required by law, the companies have submitted the confidential license agreements to the U.S. Federal Trade Commission and the U.S. Department of Justice for review. In accordance with the license agreements, the parties have terminated all ongoing Hatch-Waxman litigation between Amicus and Aurobindo and Lupin regarding Galafold® patents pending in the District Court.
The patent positions of companies like ours are generally uncertain and involve complex legal, technical, scientific, and factual questions. Our ability to maintain and solidify our proprietary position for our technology will depend on our success in promptly filing patent applications on new discoveries, and in obtaining effective claims and enforcing those claims once granted. We focus special attention on filing patent applications for formulations and delivery regimens for our products in development to further enhance our patent exclusivity for those products. We seek to protect our proprietary technology and processes, in part, by contracting with our employees, collaborators, scientific advisors, and our commercial consultants to ensure that any inventions resulting from the relationship are disclosed promptly, maintained in confidence until a patent application is filed, and preferably until publication of the patent application, and assigned to us or subject to a right to obtain a license. We do not know whether any of our owned patent applications or those patent applications that are licensed to us will result in the issuance of any patents. Our issued patents and those that may issue in the future, or those licensed to us, may be challenged, narrowed, invalidated, circumvented, or be found to be invalid or unenforceable, which could limit our ability to stop competitors from marketing related products and reduce the term of patent protection that we may have for our products. Neither we nor our licensors can be certain that we were the first to invent the inventions claimed in our owned or licensed patents or patent applications. In addition, our competitors may independently develop similar technologies or duplicate any technology developed by us and the rights granted under any issued patents may not provide us with any meaningful competitive advantages against these competitors. Furthermore, because of the extensive time required for development, testing, and regulatory review of a potential product, it is possible that any related patent may expire prior to or shortly after commencing commercialization, thereby reducing the advantage of the patent to our business and products.
We may rely, in some circumstances, on trade secrets to protect our technology. However, trade secrets are difficult to protect. We seek to protect our trade secret technology and processes, in part, by entering into confidentiality agreements with commercial partners, collaborators, employees, consultants, scientific advisors, and other contractors, and by contracting with our employees and some of our commercial consultants to ensure that any trade secrets resulting from such employment or consulting are owned by us. We also seek to preserve the integrity and confidentiality of our data and trade secrets by maintaining physical security of our premises and physical and electronic security of our information technology systems. While we have confidence in these individuals, organizations, and systems, agreements or security measures may be breached, and we may not have adequate remedies for any breach. In addition, our trade secrets may otherwise become known or be discovered independently by others. To the extent that our consultants, contractors, or collaborators use intellectual property owned by others in their work for us, disputes may arise as to the rights in related or resulting know-how and inventions.
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Collaboration and License Agreements
We have acquired rights to develop and commercialize our product candidates through licenses granted by various parties. We have certain obligations under these acquisitions or licensing agreements, including diligence obligations and payments, which are contingent upon achieving various development, regulatory and commercial milestones. Also, pursuant to the terms of some of these license agreements, when and if commercial sales of a product commence, we may be obligated to pay royalties to such third parties on net sales of the respective products.
The following summarizes our material rights and obligations under those licenses:
GlaxoSmithKline
In July 2012, as amended in November 2013, we entered into an agreement with GlaxoSmithKline ("GSK"), pursuant to which Amicus obtained global rights to develop and commercialize Galafold® as a monotherapy and in combination with ERT for Fabry disease (“Collaboration Agreement”). Under the terms of the Collaboration Agreement, GSK is eligible to receive post-approval and sales-based milestones up to $40 million, as well as tiered royalties in the mid-teens in eight major markets outside the U.S.
Dimerix Limited
On April 30, 2025, we entered into an exclusive license agreement with Dimerix for the commercialization of Dimerix' Phase 3 drug candidate, DMX-200, in the U.S. for treatment of FSGS and other indications. In exchange for these rights, we paid Dimerix an upfront payment of $30 million, which was recorded as a component of research and development expense in our Consolidated Statement of Operations. We will be obligated to pay Dimerix for certain success-based development and regulatory milestones for FSGS of up to a maximum aggregate amount of $75 million, regulatory milestones for other indications of up to a maximum aggregate amount of $40 million, commercial milestones of up to a maximum aggregate amount of $445 million, and tiered royalties of DMX-200 net sales in the U.S. ranging from the low-teens to low-twenties.
Dimerix will continue to fund and execute ACTION3, and Amicus will be responsible for submission and maintenance of the regulatory dossier in the United States, as well as all costs of commercialization activities. Additionally, Amicus will have the exclusive rights to develop DMX-200 in other future indications in the U.S. Amicus and Dimerix have formed a Joint Steering Committee to align the development and commercialization of DMX-200 in FSGS in U.S. The agreement otherwise contains terms common for an arrangement of this kind.
Manufacturing
We continue to rely on contract manufacturers to supply the active biopharmaceutical ingredients and finished goods for our products and product candidates. The active biopharmaceutical ingredients and final formulations for these products are manufactured under current Good Manufacturing Practice ("cGMP"). The components in the final formulation for each product are commonly used in other biopharmaceutical products and are well characterized ingredients. Although we rely on contract manufacturers, we have personnel with extensive manufacturing and quality experience to oversee our contract manufacturers. We have implemented appropriate controls for assuring the quality of both active biopharmaceutical ingredients and final drug products. Product specifications will be established in concurrence with regulatory bodies at the time of product registration. Our current arrangement with third-party manufacturers provide sufficient quantities of our program materials to meet anticipated clinical and commercial demands.
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Competition
Overview
The biotechnology and pharmaceutical industries are characterized by rapidly advancing technologies, intense competition, and a strong emphasis on proprietary products. In addition, several large pharmaceutical companies are increasingly focused on developing therapies for the treatment of rare diseases through organic growth, acquisitions, and partnerships. While we believe that our technologies, knowledge, experience, and scientific resources, provide us with competitive advantages, we face potential competition from many different sources, including commercial enterprises, academic institutions, government agencies, and private and public research institutions. Any product candidates that we successfully develop and commercialize will compete with both existing and new therapies that may become available in the future.
Many of our competitors may have significantly greater financial resources and expertise associated with research and development, regulatory approvals, and marketing approved products. These competitors also compete with us in recruiting and retaining qualified scientific and management personnel, as well as in acquiring technologies complementary to, or necessary for, our programs. Smaller or early-stage companies may also prove to be significant competitors, particularly through collaborative arrangements with large and established companies.
Our commercial opportunities could be reduced or eliminated if our competitors develop and commercialize products that are safer, more effective, have fewer side effects, are more convenient, and/or are less expensive than products that we may develop. In addition, our ability to compete may be affected because in some cases insurers or other third-party payors seek to encourage the use of generic products. This may have the effect of making branded products less attractive to buyers.
Major Competitors
Our major competitors include pharmaceutical and biotechnology companies in the U.S. and abroad that have approved therapies or therapies in development for lysosomal storage disorders. Other competitors are pharmaceutical and biotechnology companies that have approved therapies or therapies in development for rare diseases for which pharmacological chaperone technology, or next-generation ERT may be applicable. Additionally, we are aware of several early-stage, niche pharmaceutical, and biotechnology companies whose core business revolves around protein misfolding; however, we are not aware that any of these companies are currently working to develop products that would directly compete with ours. We are also aware of several pharmaceutical and biotechnology companies who are developing various treatments for novel ERTs and gene therapy. The key competitive factors affecting the success of our product candidates are likely to be their efficacy, safety, convenience, and price.
Any product candidates that we successfully develop and commercialize will compete with existing therapies and new therapies that may become available in the future. The following table lists our principal competitors and publicly available information on the status of their clinical-stage product offerings:
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Competitor (1)(2) | Indication | Product | Class of Product | Status | ||||||||||||||||||||||
| Sanofi | Fabry Disease | Fabrazyme® | ERT | Marketed | ||||||||||||||||||||||
| Pompe Disease | Myozyme®/ Lumizyme® | ERT | Marketed | |||||||||||||||||||||||
| Pompe Disease | Nexviazyme®/ Nexviadyme® | ERT | Marketed | |||||||||||||||||||||||
| Fabry Disease | Venglustat | Oral glucosylceramide synthase ("GCS") Inhibitor | Phase 3 | |||||||||||||||||||||||
| Takeda | Fabry Disease | Replagal® | ERT | Marketed | ||||||||||||||||||||||
| Chiesi | Fabry Disease | ELFABRIO® | ERT | Marketed | ||||||||||||||||||||||
| Idorsia | Fabry Disease | Lucerastat | Oral GCS Inhibitor | Phase 3 | ||||||||||||||||||||||
| Sangamo | Fabry Disease | Isaralgagene civaparvovec | Gene Therapy | Pivotal | ||||||||||||||||||||||
| Bayer | Pompe Disease | ACTUS-101 | Gene Therapy | Phase 1/2 | ||||||||||||||||||||||
| Maze/Shionogi | Pompe Disease | MZE001 | Oral glycogen synthase ("GYS1") Inhibitor | Phase 1/2 | ||||||||||||||||||||||
| Travere | FSGS | Sparsentan | ERA Inhibitor | Phase 3 | ||||||||||||||||||||||
| Vertex | FSGS | VX-147 | Oral Inhibitor | Phase 3 | ||||||||||||||||||||||
_____________________________
(1) Reflects commercial products and product candidates for which Investigational New Drug application ("IND") has been filed or are in
clinical development.
(2) In addition to the competitors listed in the table, we are aware of several companies pursuing early stage clinical development of gene therapies for
Fabry and Pompe disease.
Government Regulation
FDA Approval Process
In the U.S., biopharmaceutical products, including gene therapies, are subject to extensive regulation by the FDA. The Federal Food, Drug, and Cosmetic Act, Public Health Services Act, and other federal and state statutes and regulations, govern, among other things, the research, development, testing, manufacture, storage, recordkeeping, approval, labeling, promotion and marketing, distribution, post-approval monitoring and reporting, sampling, and import and export of biopharmaceutical products. Failure to comply with applicable U.S. requirements may subject a company to a variety of administrative or judicial sanctions, such as FDA refusal to file a marketing application, to issue complete response letters or to not approve pending NDAs or Biologics License Applications ("BLAs"), or to issue warning letters, untitled letters, Form 483s, product recalls, product seizures, total or partial suspension of production or distribution, injunctions, fines, civil penalties, litigation, government investigation, and criminal prosecution.
Biopharmaceutical product development in the U.S. typically involves nonclinical laboratory and animal tests, the submission to the FDA of an IND, which must become effective before clinical testing may commence, and adequate and well-controlled clinical trials to establish the safety and effectiveness of the drug for each indication for which FDA approval is sought. Satisfaction of FDA pre-market approval requirements typically takes many years and the actual time required varies substantially based upon the type, complexity, and novelty of the product or disease. Preclinical tests include laboratory evaluation of product chemistry, formulation, and toxicity, as well as animal studies to assess the characteristics, potential safety, and efficacy of the product. The conduct of the preclinical tests must comply with federal regulations and requirements including Good Laboratory Practices ("GLP"). The results of preclinical testing are submitted to the FDA as part of an IND along with other information including information about product chemistry, manufacturing and controls ("CMC"), and at least one proposed clinical trial protocol. Long-term preclinical safety evaluations, such as animal tests of reproductive toxicity and carcinogenicity, continue during the IND phase of development. Reproductive toxicity studies are required to allow inclusion of women of childbearing potential in clinical trials, whereas carcinogenicity studies are required for registration. The results of these long-term studies would eventually be described in product labeling.
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A 30-day review period after the submission and receipt of an IND is required prior to the commencement of clinical testing in humans. The IND becomes effective 30 days after its receipt by the FDA, and trials may begin at that point unless the FDA notifies the sponsor that the investigations are subject to a clinical hold.
Clinical trials usually involve the administration of the investigational new drug to healthy volunteers or patients under the supervision of a qualified investigator. Clinical trials must be conducted in compliance with applicable government regulations, Good Clinical Practice ("GCP"), as well as under protocols detailing the objectives of the trial, the parameters to be used in monitoring safety, and the effectiveness criteria to be evaluated. Each protocol involving testing on U.S. patients and subsequent protocol amendments must be submitted to the FDA as part of the IND.
The FDA may order the temporary or permanent discontinuation of a clinical trial at any time or impose other sanctions if it believes that the clinical trial is not being conducted in accordance with FDA requirements or presents an unacceptable risk to the clinical trial patients. The study protocol and informed consent information for patients in clinical trials must also be submitted to an Institutional Review Board ("IRB"), for approval. An IRB may also require the clinical trial at the site to be halted, either temporarily or permanently, for failure to comply with the IRB's requirements, or may impose other conditions.
Clinical trials to support an NDA or BLA for marketing approval are typically conducted in three sequential phases, but the phases may overlap. In Phase 1, the initial introduction of the drug into healthy human subjects or patients, the drug is tested to assess metabolism, pharmacokinetics, pharmacological actions, side effects associated with increasing doses, and, if possible, early evidence on pharmacodynamics effects and effectiveness.
Phase 2 usually involves trials in a limited patient population to determine the effectiveness of the drug for a particular indication or indications, dosage tolerance, and optimum dosage, and identify common adverse effects and safety risks. If a compound demonstrates evidence of efficacy and an acceptable safety profile in Phase 2 evaluations, Phase 3 trials are undertaken to obtain the additional information about clinical efficacy and safety in a larger number of patients over longer treatment periods, typically at geographically dispersed clinical trial sites, to permit the FDA to evaluate the overall benefit-risk relationship of the drug and to provide adequate information for the labeling of the drug.
The FDA has established the Office of Tissue and Advanced Therapies within the Center for Biologics Evaluation and Research, or CBER, to consolidate the review of gene therapy and related products, and has established the Cellular, Tissue and Gene Therapies Advisory Committee to advise CBER in its review.
In addition to the regulations discussed above, there are a number of additional standards that apply to clinical trials involving gene therapies. The FDA has issued various guidance documents regarding gene therapies, which outline additional factors that the FDA will consider at each of the above stages of development and relate to, among other things: the proper preclinical assessment of gene therapies; the use of innovative clinical trial designs to expedite development, including single-arm trials utilizing participants as their own controls and externally controlled studies utilizing historical or real-world data as the comparator group; the CMC information that should be included in an IND application; the proper design of tests to measure product potency in support of an IND or BLA application; and measures to observe delayed adverse effects in subjects who have been exposed to investigational gene therapies when the risk of such effects is high. Further, the FDA usually recommends that sponsors observe subjects for potential gene therapy-related delayed adverse events for a 15-year period, including a minimum of five years of annual examinations followed by 10 years of annual queries, either in person or by questionnaire. NIH and the FDA have a publicly accessible database, the Genetic Modification Clinical Research Information System, which includes information on gene therapy trials and serves as an electronic tool to facilitate the reporting and analysis of adverse events on these trials.
With respect to CMC requirements for cell and gene therapies, the FDA takes a more flexible, risk-based approach, aimed at expediting development of such treatments while maintaining product quality. The FDA recently clarified its expectations in a January 2026 announcement. While statutory and current Good Manufacturing Practices (“cGMP”) standards remain unchanged, the timing and depth of CMC expectations will be better tailored to the realities of cell and gene therapy development. The FDA indicated it will accept more phase‑appropriate CMC packages during clinical development, allow greater flexibility in setting initial commercial specifications (with plans to refine them post‑approval), and move away from rigid expectations such as a default “three PPQ lot” standard for process validation.
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After completion of the required clinical testing, an NDA or BLA is prepared and submitted to the FDA for the determination of efficacy and safety. FDA approval of the NDA or BLA is required before marketing of the product may begin in the U.S. The NDA or BLA must include the results of all preclinical, clinical, and other testing and a compilation of data relating to the product's pharmacology, chemistry, manufacture, and controls. The cost of preparing and submitting an NDA or BLA is substantial. Under federal law, the submission of most NDAs and BLAs is additionally subject to a substantial application user fee; although for orphan drugs these fees are waived, and the holder of an approved NDA or BLA may also be subject to annual product and establishment user fees. These fees are typically increased annually.
The FDA has 60 days from its receipt of an NDA or BLA to determine whether the application will be accepted for filing based on the agency's threshold determination that it is sufficiently complete to permit substantive review. Once the submission is accepted for filing, the FDA begins an in-depth review. The FDA has agreed to certain performance goals in the review of NDAs and BLAs. Marketing applications are assigned review status during the filing period. Review status could be either standard or priority. Most such applications for standard review are reviewed within 12 months under the Prescription Drug User Fee Act ("PDUFA") (two months for filing plus ten months for review). The FDA attempts to review a drug candidate that is eligible for priority review within six months, as discussed below. The review process may be extended by the FDA for three additional months to evaluate major amendments submitted during the pre-specified PDUFA review clock. Also, delays may occur due to changing policies, shifting priorities, and recent staffing cuts at the FDA. The FDA may also refer applications for novel drug products or drug products which present difficult questions of safety or efficacy to an advisory committee for public review, typically a panel that includes clinicians and other experts, for review, evaluation, and a recommendation as to whether the application should be approved. The FDA is not bound by the recommendation of an advisory committee, but it generally follows such recommendations. Before approving an NDA or BLA, the FDA will typically inspect one or more clinical sites to assure compliance with GCP. Additionally, the FDA will inspect the facility or the facilities at which the drug is manufactured. The FDA may also undertake an audit of nonclinical and clinical trial sites. The FDA will not approve the product candidate unless compliance with cGMP is satisfactory and the NDA or BLA contains data that provide substantial evidence that the drug is safe and effective in the indication studied and to be marketed. During the product approval process, the FDA also will determine whether a risk evaluation and mitigation strategy, or REMS, is necessary to assure the safe use of the product candidate. A REMS could include medication guides, physician communication plans and elements to assure safe use, such as restricted distribution methods, patient registries, and other risk minimization tools. If the FDA concludes a REMS is needed, the sponsor of the NDA or BLA must submit a proposed REMS; the FDA will not approve the NDA or BLA without a REMS, if required.
After the FDA evaluates the NDA or BLA and the manufacturing facilities, it issues an approval letter or a complete response letter. Complete response letters outline the deficiencies in the submission that prevent approval and may require substantial additional testing or information for the FDA to reconsider the application. If and when those deficiencies have been addressed to the FDA's satisfaction in an amendment submitted to the NDA or BLA, the FDA will then issue an approval letter. The FDA has committed to reviewing such resubmissions in two or six months depending on the type and extent of information included.
An approval letter authorizes commercial marketing of the drug with specific prescribing information for specific indications. As a condition of NDA or BLA approval, the FDA may require substantial post-approval commitments or requirements to conduct additional testing and/or surveillance to monitor the drug's safety or efficacy and may impose other conditions, including distribution and labeling restrictions which can materially affect the potential market and profitability of the drug. Once granted, product approvals may be withdrawn if compliance with regulatory standards is not maintained, problems are identified following initial marketing, or post-marketing commitments are not met. In September 2025, the FDA issued draft guidance on the importance of post-approval safety and efficacy data for gene therapies. The FDA acknowledged that approval of gene therapies is often based on data from relatively small clinical trials of short duration since large, long-term randomized trials are generally not feasible. As a result, key questions about durability of treatment effect and long-term safety remain at the time of approval. The draft guidance provides methods and data sources sponsors can use to support ongoing safety and efficacy evaluation, including use of real-world evidence, electronic health records, registries, and decentralized data collection.
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The Hatch-Waxman Act
In seeking approval for a drug through an NDA, applicants are required to list with the FDA certain patent(s) with claims that cover the applicant's product or approved method of use. Upon approval of a drug, each of the patents listed in the application for the drug is then published in the FDA's Approved Drug Products with Therapeutic Equivalence Evaluations, commonly known as the Orange Book. Drugs listed in the Orange Book can, in turn, be cited by potential competitors in support of approval of an ANDA. An ANDA provides for marketing of a drug product that has the same route of administration, active ingredients strength, and dosage form as the listed drug and has been shown through bioequivalence testing to be, in most cases, therapeutically equivalent to the listed drug. ANDA applicants are not required to conduct or submit results of preclinical or clinical tests to prove the safety or effectiveness of their drug product, other than the requirement for bioequivalence testing. Drugs approved in this way are commonly referred to as "generic equivalents" to the listed drug and can often be substituted by pharmacists under prescriptions written for the original listed "innovator" drug.
The ANDA applicant is required to certify to the FDA concerning any patents listed for the approved product in the FDA's Orange Book. Specifically, the applicant must certify that: (i) the required patent information has not been filed; (ii) the listed patent has expired; (iii) the listed patent has not expired but will expire on a particular date and approval is sought after patent expiration; or (iv) the listed patent is invalid or will not be infringed by the new product. A certification that the new product will not infringe the already approved product's listed patents or that such patents are invalid is called a Paragraph 4 certification. If the applicant does not challenge the listed patents, the ANDA application will not be approved until all the listed patents claiming the referenced product have expired.
If the ANDA applicant submits a Paragraph 4 certification to the FDA, the applicant must also send notice of the Paragraph 4 certification to the NDA and patent holders once the ANDA has been accepted for filing by the FDA. The NDA and patent holders may then initiate a patent infringement lawsuit in response to the notice of the Paragraph 4 certification. The filing of a patent infringement lawsuit within 45 days of the receipt of a Paragraph 4 certification automatically prevents the FDA from approving the ANDA until the earlier of 30 months, expiration of the patent, settlement of the lawsuit or a decision in the infringement case that is favorable to the ANDA applicant.
Patent term and data exclusivity run in parallel. An ANDA application also will not be approved until any non-patent exclusivity, such as exclusivity for obtaining approval of an NCE, listed in the Orange Book for the referenced product has expired ("New Chemical Entity Market Exclusivity"). Federal law provides a period of five years following approval of a drug containing no previously approved active ingredients, during which ANDAs for generic versions of those drugs cannot be submitted unless the submission contains a Paragraph 4 certification that challenges a listed patent, in which case the submission may be made four years following the original product approval.
Federal law provides for a period of three years of exclusivity following approval of a listed drug that contains previously approved active ingredients but is approved in a new dosage form, route of administration or combination, or for a new use, the approval of which was required to be supported by new clinical trials conducted by or for the sponsor, during which the FDA cannot grant effective approval of an ANDA based on that listed drug for the same new dosage form, route of administration or combination, or new use.
Other Regulatory Requirements
Once an NDA or BLA is approved, a product will be subject to certain post-approval requirements. For instance, the FDA closely regulates the post-approval marketing and promotion of drugs, including standards and regulations for direct-to-consumer advertising, communications regarding unindicated uses, industry-sponsored scientific and educational activities, and promotional activities involving the internet. Products approved under Subpart H or Subpart E carry additional post-marketing considerations and requirements.
Drugs may be promoted only for approved indications and in accordance with the provisions of the approved labeling. Changes to some of the conditions established in an approved application, including changes in indications, new safety information, labeling, or manufacturing processes or facilities, require submission and FDA approval of a new NDA, NDA supplement, BLA, or BLA supplement before the change can be implemented. New efficacy claims require submission and approval of an NDA supplement and BLA supplement for each new indication.
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The efficacy claims typically require new clinical data similar to those included in the original application. The FDA uses the same procedures and actions in reviewing NDA and BLA supplements as it does in reviewing NDAs and BLAs. Additional exclusivity may be granted for new efficacy claims. Generic ANDAs cannot be labeled for these types of claims until the new exclusivity period expires.
Adverse event reporting and submission of periodic reports is required following FDA approval of an NDA or BLA. The FDA also may require post-marketing testing, known as Phase 4 testing, risk evaluation and mitigation strategies, and surveillance to monitor the effects of an approved product, or place conditions on an approval that could restrict the distribution or use of the product. In addition, quality control as well as drug manufacture, packaging, and labeling procedures must continue to conform to cGMP, after approval. Drug manufacturers and certain subcontractors are required to register their establishments with FDA and certain state agencies and are subject to routine inspections by the FDA during which the agency inspects manufacturing facilities to access compliance with cGMP. Accordingly, manufacturers must continue to expend time, money, and effort in the areas of production and quality control to maintain compliance with cGMP. Regulatory authorities may withdraw product approvals or request product recalls if a company fails to comply with regulatory standards, if it encounters problems following initial marketing, or if previously unrecognized problems are subsequently discovered.
Orphan Drugs
Under the Orphan Drug Act, the FDA may grant orphan drug designation to drugs intended to treat a rare disease or condition, which is generally a disease or condition that affects fewer than 200,000 individuals in the U.S. Orphan drug designation must be requested before submitting an NDA or BLA. After the FDA grants orphan drug designation, the generic identity of the drug and its potential orphan use are disclosed publicly by the FDA. Orphan drug designation does not convey any advantage in or shorten the duration of the regulatory review and approval process. The first NDA or BLA applicant with FDA orphan drug designation for a particular active ingredient to receive FDA approval of the designated drug for the disease indication for which it has such designation, is entitled to a seven-year exclusive marketing period ("Orphan Drug Exclusivity") in the U.S. for that product, for that indication. During the seven-year period, the FDA may not finally approve any other applications to market the same drug for the same disease, except in limited circumstances, such as a showing of clinical superiority to the product with orphan drug exclusivity or if the license holder cannot supply sufficient quantities of the product. Orphan drug exclusivity does not prevent the FDA from approving a different drug for the same disease or condition, or the same drug for a different disease or condition, provided that the sponsor has conducted appropriate clinical trials required for approval. Among the other benefits of orphan drug designation are tax credits for certain research and a waiver of the NDA or BLA application user fee for the orphan indication.
Pediatric Information
Under the Pediatric Research Equity Act of 2007 ("PREA"), NDAs or supplements to NDAs and BLAs or supplements to BLAs must contain data to assess the safety and effectiveness of the drug for the claimed indications in all relevant pediatric subpopulations and to support dosing and administration for each pediatric subpopulation for which the drug is safe and effective. The FDA may grant deferrals for submission of data or full or partial waivers. Unless otherwise required by regulation, PREA does not apply to any drug for an indication for which orphan designation has been granted.
Fast Track Designation
Under the Fast Track program, the sponsor of an IND may request the FDA to designate the drug candidate as a Fast Track drug if it is intended to treat a serious condition and fulfill an unmet medical need. The FDA must determine if the drug candidate qualifies for Fast Track designation within 60 days of receipt of the sponsor's request. Once the FDA designates a drug as a Fast Track candidate, it is required to facilitate the development and expedite the review of that drug by providing more frequent communication with and guidance to the sponsor.
In addition to other benefits such as greater interactions with the FDA, the FDA may initiate review of sections of a Fast Track drug's NDA or BLA before the application is complete. This rolling review is available if the applicant provides, and the FDA approves, a schedule for the submission of the remaining information and the applicant pays applicable user fees. However, the FDA's review period as specified under PDUFA for filing and reviewing an application does not begin until the last section of the NDA or BLA has been submitted. Additionally, the Fast Track designation may be withdrawn by the FDA if the FDA believes that the designation is no longer supported by data emerging in the clinical trial process.
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Breakthrough Therapy Designation
Breakthrough Therapy designation is intended to expedite the development and review of a candidate that is planned for use to treat a serious or life-threatening disease or condition when preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints. A Breakthrough Therapy designation conveys all of the Fast Track program features, as well as more intensive FDA guidance on an efficient drug development program. The FDA also has an organizational commitment to involve senior management in such guidance.
Priority Review
Under FDA policies, a drug candidate is eligible for priority review, or review within six months from filing for a new molecular entity ("NME") or six months from submission for a non-NME if the drug candidate provides a significant improvement compared to marketed drugs in the treatment, diagnosis, or prevention of a disease, rather than the standard review of ten months under current PDUFA guidelines. A Fast Track designated drug candidate would ordinarily meet the FDA's criteria for priority review. The FDA makes its determination of priority or standard review during the 60-day filing period after an initial NDA or BLA submission.
Accelerated Approval
Under the FDA's accelerated approval regulations, the FDA may approve a drug for a serious or life-threatening illness that provides meaningful therapeutic benefit to patients over existing treatments based upon a surrogate endpoint that is reasonably likely to predict clinical benefit. This approval mechanism is provided for under 21CRF314 Subpart H and Subpart E. In this case, clinical trials are conducted in which a surrogate endpoint is used as the primary outcome for approval. A surrogate endpoint is reasonably likely to predict clinical benefit, or an effect on a clinical endpoint that can be measured earlier than an effect on irreversible morbidity or mortality, that is reasonably likely to predict an effect on irreversible morbidity or mortality or other clinical benefit, taking into account the severity, rarity, or prevalence of the condition and the availability or lack of alternative treatments. This surrogate endpoint substitutes for a direct measurement of how a patient feels, functions, or survives and is considered reasonably likely to predict clinical benefit. Such surrogate endpoints may be measured more easily or more rapidly than clinical endpoints. A drug candidate approved on this basis is subject to rigorous post-marketing compliance requirements, including the completion of Phase 4 or post-approval clinical trials to confirm the effect on the clinical endpoint. Under the Food and Drug Omnibus Reform Act of 2022 (“FDORA”), the FDA is now permitted to require, as appropriate, that such trials be underway prior to approval or within a specific time period after the date of approval for a product granted accelerated approval. When the Phase 4 commitment is successfully completed, the biomarker is deemed to be a surrogate endpoint. Failure to conduct required post-approval studies or confirm a clinical benefit during post-marketing studies, could lead the FDA to withdraw the drug from the market on an expedited basis. All promotional materials for drug candidates approved under accelerated regulations are subject to prior review by the FDA.
FDA Commissioner’s National Priority Review Voucher ("CNPV") Pilot Program
In June 2025, the FDA announced the CNPV pilot program, which was designed to accelerate the development and review of certain drugs and biologics that are aligned with U.S. national health priorities, such as addressing a U.S. public health crisis, developing more innovative cures for the American people, addressing a large unmet medical need, onshoring drug development and manufacturing to advance the health interests of Americans and strengthen U.S. supply chain resiliency, and increasing affordability. The FDA has stated that voucher recipients will receive a decision with respect to an application on an accelerated basis, as well as enhanced communication with review staff throughout the review process. The FDA expects the CNPV program to accelerate the application review timeline from 10-12 months to 1-2 months by convening a multidisciplinary team of physicians and scientists for a team-based review, interacting frequently with the sponsor of the application to clarify questions, and completing review of the application concurrently. The FDA retains full discretion to extend the review window if the data or application components submitted are insufficient or incomplete, if the results of the pivotal trial(s) are ambiguous, or if the review is particularly complex. The FDA has indicated the CNPV program does not change the FDA’s rigorous safety and efficacy standards for review and approval. In late 2025, the FDA began issuing approvals for drugs with CNPVs within the 1-2 month review timeframe. The CNPV program is new, limited in scope, and subject to evolving guidance and available FDA resources. The FDA retains broad discretion to modify the criteria, processes, or benefits of the program and may rescind participation or alter timelines or the intended benefits at any time. Adding to the uncertainty, concerns have been raised regarding the legality of the CNPV program.
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Section 505(b)(2) New Drug Applications
Most drug products obtain FDA marketing approval pursuant to an NDA, an ANDA, or a BLA. A fourth alternative is a special type of NDA, commonly referred to as a Section 505(b)(2) NDA, which enables the applicant to rely, in part, on the safety and efficacy data of an existing product, or published literature, in support of its application.
505(b)(2) NDAs often provide an alternate path to FDA approval for new or improved formulations or new uses of previously approved products. Section 505(b)(2) permits the submission of an NDA for which at least some of the information required for approval comes from studies not conducted by or for the applicant and for which the applicant has not obtained a right of reference. The applicant may rely upon certain preclinical or clinical studies conducted for an approved product. The FDA may also require companies to perform additional studies or measurements to support the change from the approved product. The FDA may then approve the new product candidate for all or some of the label indications for which the referenced product has been approved, as well as for any new indication sought by the Section 505(b)(2) applicant.
To the extent that the Section 505(b)(2) applicant is relying on studies conducted for an already-approved product, the applicant is required to certify to the FDA concerning any patents listed for the approved product in the Orange Book to the same extent as an ANDA applicant. Thus approval of a 505(b)(2) NDA can be stalled until all the listed patents claiming the referenced product have expired, until any non-patent exclusivity, such as exclusivity for obtaining approval of an NCE, listed in the Orange Book for the referenced product has expired, and, in the case of a Paragraph 4 certification and subsequent patent infringement suit, until the earlier of 30 months, settlement of the lawsuit or a decision in the infringement case that is favorable to the Section 505(b)(2) applicant.
Biologics Price Competition and Innovation Act
The Biologics Price Competition and Innovation Act of 2009 ("BPCIA"), which was enacted as part of the Patient Protection and Affordable Care Act of 2010, as amended by the Health Care and Education Reconciliation Act of 2010 ("PPACA") created an abbreviated approval pathway for biological products that are demonstrated to be "biosimilar" or "interchangeable" with an FDA-licensed reference biological product via an approved BLA. Biosimilarity to an approved reference product requires that there be no differences in conditions of use, route of administration, dosage form, and strength, and no clinically meaningful differences between the biological product and the reference product in terms of safety, purity, and potency. Biosimilarity is demonstrated in steps beginning with rigorous analytical studies or "fingerprinting", in vitro studies, in vivo animal studies, and generally at least one clinical study, absent a waiver from the Secretary of Health and Human Services. The biosimilarity exercise tests the hypothesis that the investigational product and the reference product are the same. If at any point in the stepwise biosimilarity process a significant difference is observed, then the products are not biosimilar, and the development of a stand-alone NDA or BLA is necessary. In order to meet the higher hurdle of interchangeability, a sponsor must demonstrate that the biosimilar product can be expected to produce the same clinical result as the reference product, and for a product that is administered more than once, that the risk of switching between the reference product and biosimilar product is not greater than the risk of maintaining the patient on the reference product. Complexities associated with the larger, and often more complex, structures of biological products, as well as the process by which such products are manufactured, pose significant hurdles to implementation that are still being evaluated by the FDA. Under an October 2025 draft guidance, the FDA will no longer routinely require data from comparative clinical efficacy studies to demonstrate biosimilarity for therapeutic protein products. Adequate data from comparative analytic, pharmacokinetic, and immunogenicity assessments may suffice to support approval of a biosimilar. Under the BPCIA, a reference biologic is granted 12 years of exclusivity from the time of first licensure of the reference product.
Anti-Kickback, False Claims Laws, the Prescription Drug Marketing Act and Other Regulations
Our activities are subject to various federal and state fraud and abuse laws, including, without limitation, the federal Anti-Kickback Statute, the federal civil False Claims Act, and laws and regulations pertaining to limitations on and reporting of healthcare provider payments (physician sunshine laws). These laws and regulations are interpreted and enforced by various federal, state and local authorities including CMS, the Office of Inspector General for the U.S. Department of Health and Human Services, the U.S. Department of Justice, individual U.S. Attorney offices within the Department of Justice, and state and local governments. These laws include:
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•the U.S. federal Anti-Kickback Statute, which prohibits, among other things, persons or entities from knowingly and willfully soliciting, offering, receiving or paying any remuneration, directly or indirectly, overtly or covertly, in cash or in kind, to induce or reward either the referral of an individual for, or the purchase, lease, order, or arranging for or recommending the purchase, lease or order of, any good or service, for which payment may be made, in whole or in part, under federal healthcare programs such as Medicare and Medicaid. A person or entity does not need to have actual knowledge of the statute or specific intent to violate it in order to have committed a violation;
•the U.S. civil False Claims Act (which can be enforced through “qui tam,” or whistleblower actions, by private citizens on behalf of the federal government), prohibits any person from, among other things, knowingly presenting, or causing to be presented false or fraudulent claims for payment of government funds or knowingly making, using or causing to be made or used, a false record or statement material to an obligation to pay money to the government or knowingly and improperly avoiding, decreasing or concealing an obligation to pay money to the U.S. federal government;
•U.S. federal Health Insurance Portability and Accountability Act of 1996, or HIPAA, which imposes criminal liability and amends provisions on the reporting, investigation, enforcement, and penalizing of civil liability for, among other things, knowingly and willfully executing, or attempting to execute, a scheme to defraud any healthcare benefit program, or knowingly and willfully falsifying, concealing or covering up a material fact or making any materially false statement, in connection with the delivery of or payment for healthcare benefits, items or services by a healthcare benefit program, which includes both government and privately funded benefits programs; similar to the U.S. federal Anti-Kickback Statute, a person or entity does not need to have actual knowledge of the statute or specific intent to violate it in order to have committed a violation;
•state laws and regulations, including state anti-kickback and false claims laws, that may apply to our business practices, including but not limited to, research, distribution, sales and marketing arrangements and claims involving healthcare items or services reimbursed by any third-party payer, including private insurers; state laws that require pharmaceutical companies to comply with the pharmaceutical industry’s voluntary compliance guidelines and the relevant compliance guidance promulgated by the U.S. federal government, or otherwise restrict payments that may be made to healthcare providers and other potential referral sources; and state laws and regulations that require drug manufacturers to file reports relating to pricing and marketing information, which requires tracking gifts and other remuneration and items of value provided to healthcare professionals and entities; and
•the Physician Payments Sunshine Act, implemented as the Open Payments program, and its implementing regulations, requires certain manufacturers of drugs, devices, biologics and medical supplies that are reimbursable under Medicare, Medicaid, or the Children’s Health Insurance Program to report annually to CMS information related to certain payments made in the preceding calendar year and other transfers of value to physicians and teaching hospitals, as well as ownership and investment interests held by physicians and their immediate family members; beginning in 2022, applicable manufacturers are required to report such information regarding payments and transfers of value provided, as well as ownership and investment interests held, during the previous year to physician assistants, nurse practitioners, clinical nurse specialists, certified nurse anesthetists, and certified nurse-midwives.
Violations of any of these laws or any other governmental regulations that may apply to us, may subject us to significant civil, criminal and administrative sanctions including penalties, damages, fines, imprisonment, and exclusion from government funded healthcare programs, such as Medicare and Medicaid, and/or adverse publicity. Moreover, government entities and private litigants have asserted claims under state consumer protection statutes against pharmaceutical and medical device companies for alleged false or misleading statements in connection with the marketing, promotion and/or sale of pharmaceuticals.
Physician Drug Samples
As part of the sales and marketing process, pharmaceutical companies frequently provide samples of approved drugs to physicians. The Prescription Drug Marketing Act (the "PDMA") imposes requirements and limitations upon the provision of drug samples to physicians, as well as prohibits states from licensing distributors of prescription drugs unless the state licensing program meets certain federal guidelines that include minimum standards for storage, handling, and record keeping. In addition, the PDMA sets forth civil and criminal penalties for violations.
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Regulation Outside the U.S.
In addition to regulations in the U.S., we are subject to a variety of regulations in other jurisdictions governing clinical studies, commercial sales, and distribution of our products. Most countries outside the U.S. require that clinical trial applications be submitted to and approved by the local regulatory authority for each clinical study. In addition, whether or not we obtain FDA approval for a product, we must obtain approval of a product by the comparable regulatory authorities of countries outside the U.S. before we can commence clinical studies or marketing of the product in those countries. The approval process varies from country to country, and the time may be longer or shorter than that required for FDA approval.
To obtain regulatory approval of an orphan drug under the E.U. regulatory system, we are mandated to submit a marketing authorization application ("MAA") to be assessed in the centralized procedure. The centralized procedure, which came into operation in 1995, allows applicants to obtain a marketing authorization that is valid throughout the E.U. It is compulsory for medicinal products manufactured using biotechnological processes, for orphan medicinal products and for human products containing a new active substance which was not authorized in the community before 20 May 2004 (date of entry into force of Regulation (EC) No 726/2004) and which are intended for the treatment of AIDS, cancer, neurodegenerative disorder, or diabetes. The centralized procedure is optional for any other products containing new active substances not authorized in the community before 20 May 2004 or for products which constitute a significant therapeutic, scientific, or technical innovation or for which a community authorization is in the interests of patients at community level. When a company wishes to place on the market a medicinal product that is eligible for the centralized procedure, it sends an application directly to the European Medicines Agency ("EMA"), to be assessed by the Committee for Medicinal Products for Human Use ("CHMP"). The procedure results in a commission decision, which is valid in all E.U. member states. Centrally-authorized products may be marketed in all member states. Under the centralized procedure, full copies of the MAA are sent to a rapporteur and a co-rapporteur designated by the competent EMA scientific committee. They coordinate the EMA's scientific assessment of the medicinal product and prepare draft reports. Once the draft reports are prepared (other experts might be called upon for this purpose), they are sent to the CHMP, whose comments or objections are communicated to the applicant. The rapporteur is therefore the privileged interlocutor of the applicant and continues to play this role, even after the MAA has been granted approval.
The rapporteur and co-rapporteur then assess the applicant's replies, submit them for discussion to the CHMP and, taking into account the conclusions of this debate, prepare a final assessment report. Once the evaluation is completed, the CHMP gives a favorable or unfavorable opinion as to whether to grant the authorization. When the opinion is favorable, it shall include the draft summary of the product's characteristics, the package leaflet and the texts proposed for the various packaging materials. The time limit for the evaluation procedure is 210 days. The EMA then has fifteen days to forward its opinion to the commission. This is the start of the second phase of the procedure: the decision-making process. The Agency sends to the commission its opinion and assessment report, together with annexes containing: the SmPC ("Annex 1"); the particulars of the Marketing Authorization Holder ("MAH") responsible for batch release, the particulars of the manufacturer of the active substance and the conditions of the marketing authorization ("Annex 2"); and the labelling and the package leaflet ("Annex 3"). The annexes are translated into the 22 other official languages of the E.U. During the decision-making process, the commission services verify that the marketing authorization complies with Union law. The commission has fifteen days to prepare a draft decision. The medicinal product is assigned a community registration number, which will be placed on its packaging if the marketing authorization is granted. During this period, various commission directorates-general are consulted on the draft marketing authorization decision.
The draft decision is then sent to the Standing Committee on Medicinal Products for Human Use, (member states have one representative each in both of these committees) for their opinions. The centralized procedure provides for the grant of a single marketing authorization that is valid for all E.U. member states. The "decentralized procedure" provides for approval by one or more other, or concerned, member states of an assessment of an application performed by one member state, known as the reference member state. Under this procedure, an applicant submits an application, or dossier, and related materials including a draft summary of product characteristics, and draft labeling and package leaflet, to the reference member state and concerned member states. The reference member state prepares a draft assessment and drafts of the related materials within 120 days after receipt of a valid application. Within 90 days of receiving the reference member state's assessment report, each concerned member state must decide whether to approve the assessment report and related materials. If a member state cannot approve the assessment report and related materials on the grounds of potential serious risk to the public health, the disputed points may eventually be referred to the European Commission, whose decision is binding on all member states.
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We have obtained an orphan medicinal product designation in the E.U. from the EMA for Galafold® for the treatment of Fabry disease and the combination product, Pombiliti® + Opfolda®, for the treatment of Pompe disease. Applications from persons or companies seeking "orphan medicinal product designation" for products they intend to develop for the diagnosis, prevention, or treatment of life-threatening or very serious conditions that affect not more than 5 in 10,000 persons in the E.U. are reviewed by the Committee for Orphan Medicinal Products ("COMP"). In addition, orphan drug designation can be granted if the drug is intended for a life threatening, seriously debilitating, or serious and chronic condition in the E.U. and that without incentives it is unlikely that sales of the drug in the E.U. would be sufficient to justify developing the drug. Orphan drug designation is only available if there is no other satisfactory method approved in the E.U. of diagnosing, preventing, or treating the condition, or if such a method exists, the proposed orphan drug will be of significant benefit to patients.
Orphan drug designation provides opportunities for fee reductions, protocol assistance and access to the centralized procedure before and during the first year after marketing approval. Fee reductions are not limited to the first year after marketing approval for small and medium enterprises. In addition, if a product which has an orphan drug designation subsequently receives EMA marketing approval for the indication for which it has such designation, the product is entitled to orphan market exclusivity, which means the EMA may not approve any other application to market a similar drug for the same indication for a period of 10 years. The exclusivity period may be reduced to six years if the designation criteria are no longer met, including where it is shown that the product is sufficiently profitable not to justify maintenance of market exclusivity. Competitors may receive marketing approval of different drugs or biologics for the indications for which the orphan product has exclusivity. In order to do so, however, they must demonstrate that the new drugs or biologics are clinically superior over the existing orphan product. This demonstration of clinical superiority may be done at the time of initial approval or in post-approval studies, depending on the type of marketing authorization granted.
In March 2016, the EMA launched an initiative, the Priority Medicines (“PRIME”) scheme, to facilitate development of product candidates in indications, often rare, for which few or no therapies currently exist. The PRIME scheme is intended to encourage drug development in areas of unmet medical need and provides accelerated assessment of products representing substantial innovation reviewed under the centralized procedure. Many benefits accrue to sponsors of product candidates with PRIME designation, including but not limited to, early and proactive regulatory dialogue with the EMA, frequent discussions on clinical trial designs and other development program elements, and accelerated MAA assessment once a dossier has been submitted. Importantly, a dedicated contact and rapporteur from the CHMP is appointed early in the PRIME scheme facilitating increased understanding of the product at EMA’s committee level. An initial meeting initiates these relationships and includes a team of multidisciplinary experts at the EMA to provide guidance on the overall development and regulatory strategies.
We have obtained a positive opinion for our pediatric investigation plan ("PIP") in the E.U. for Galafold® for the treatment of Fabry disease as well. A PIP is a development plan aimed at ensuring that the necessary data are obtained to support the authorization of a medicine for children, through studies in children. All applications for marketing authorization for new medicines have to include the results of studies as described in an agreed PIP, unless the medicine is exempt because of a deferral or waiver. This requirement also applies when a marketing-authorization holder wants to add a new indication, pharmaceutical form, or route of administration for a medicine that is already authorized and covered by intellectual property rights. Several rewards and incentives for the development of pediatric medicines for children are available in the E.U. Medicines authorized across the E.U. with the results of studies from a PIP included in the product information are eligible for an extension of their supplementary protection certificate by six months. This is the case even when the studies' results are negative. For orphan medicines, the incentive is an additional two years of market exclusivity. Scientific advice and protocol assistance at the agency are free of charge for questions relating to the development of pediatric medicines. Medicines developed specifically for children that are already authorized but are not protected by a patent or supplementary protection certificate are eligible for a pediatric-use marketing authorization ("PUMA"). If a PUMA is granted, the product will benefit from 10 years of market protection as an incentive.
Effective January 1, 2021, following the U.K. exit of the E.U., the MHRA is the U.K.’s standalone medicines and medical devices regulator. As a result of the Northern Ireland protocol, different rules apply in Northern Ireland than in England, Wales and Scotland (together Great Britain, "GB"); broadly, Northern Ireland continues to follow the E.U. regulatory regime, but its national competent authority remains the MHRA. The MHRA has published a draft guidance outlining the various aspects of the U.K. regulatory regime for medicines in GB and in Northern Ireland. The guidance includes clinical trials, marketing authorizations, importing, exporting and pharmacovigilance and is relevant to any business involved in the research, development or commercialization of medicines in the U.K. The new guidance has been given effect via the Human Medicines Regulations ("Amendment etc.") ("E.U. Exit") Regulations 2019 (the "Exit Regulations"). The U.K. regulatory regime largely mirrors that of the E.U.
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The MHRA has introduced changes to national licensing procedures, including procedures to prioritize access to new medicines that will benefit patients, an accelerated assessment procedure and new routes of evaluation for novel products and biotechnological products. All existing E.U. marketing authorizations ("MAs") for centrally authorized products were automatically converted ("grandfathered") into U.K. MAs free-of-charge on January 1, 2021. Amicus has completed the necessary baseline submission for conversion and was granted Marketing Authorization on August 4, 2021 for Galafold® with an effective date of January 1, 2021.
There is no pre-marketing authorization orphan designation. Instead, the MHRA reviews applications for orphan designation in parallel to the corresponding MA application. The criteria are essentially the same, but have been tailored for the GB market, e.g. the prevalence of the condition in GB (rather than the E.U.) must not be more than 5 in 10,000. Should an orphan designation be granted, the period or market exclusivity will be set from the date of first approval of the product in GB or E.U./European Economic Area, wherever is earliest. Galafold® and Pombiliti® + Opfolda® have been granted orphan designation by the MHRA.
The PIP application process for applicants is simplified by offering an expedited assessment where possible, and by mirroring the submission format, content and terminology of the E.U.-PIP system. The MHRA is taking decisions on PIP and waiver opinions, modifications and compliance statements to support pediatric market authorization decisions, while acknowledging that Northern Ireland continues to be part of the E.U.’s system for pediatric medicines development including agreement of E.U. PIPs or waivers.
The MHRA has maintained the early access to medicine scheme ("EAMS"). EAMS is designed to give patients with life threatening or seriously debilitating conditions access to medicines that do not yet have a marketing authorization when there is a clear unmet medical need. Medicines with a positive EAMS opinion could be made available to patients 12-18 months ahead of formal marketing authorization. As the initial step in this process, the applicant must apply for and be granted a Promising Innovative Medicine (“PIM”) designation. The designation is issued after an MHRA scientific designation meeting on the basis of non-clinical and clinical data available on the product, in a defined disease area. Following designation, the applicant is expected to complete a clinical development program within a reasonable time period, in order to continue with an application under the EAMS.
We have obtained orphan drug designation in Japan for migalastat for the treatment of Fabry Disease. We also have other Orphan Drug applications approved in other world markets including Switzerland, Australia, South Korea and Taiwan. The Ministry of Health, Labor, and Welfare, based on the opinion of the Pharmaceutical Affairs and Food Sanitation Council, grants orphan status to drugs intended to address serious illnesses with high unmet medical need that affect fewer than 50,000 patients in Japan. In 2020, orphan drug designation was granted in Japan for AT-GAA for the treatment of Pompe disease. Orphan designation provides certain benefits and incentives, including priority review for marketing authorization and a period of 10 years of market exclusivity if the drug candidate is approved for the designated indication.
U.S. Foreign Corrupt Practices Act
The Foreign Corrupt Practices Act, or the FCPA, generally prohibits offering, promising, giving, or authorizing others to give anything of value, either directly or indirectly, to a non-U.S. government official in order to influence official action, or otherwise obtain or retain business. The FCPA also requires public companies to make and keep books and records that accurately and fairly reflect the transactions of the corporation and to devise and maintain an adequate system of internal accounting controls. Our industry is heavily regulated and therefore involves significant interaction with public officials, including officials of non-U.S. governments. Additionally, in many other countries, the health care providers who prescribe pharmaceuticals are employed by their government, and the purchasers of pharmaceuticals are government entities; therefore, our dealings with these prescribers and purchasers are subject to regulation under the FCPA. Recently, the Securities and Exchange Commission ("SEC") and Department of Justice have increased their FCPA enforcement activities with respect to pharmaceutical companies. Violations could result in fines, criminal sanctions against us, our officers, or our employees, the closing down of our facilities, requirements to obtain export licenses, cessation of business activities in sanctioned countries, implementation of compliance programs, and prohibitions on the conduct of our business. Enforcement actions may be brought by the Department of Justice or the SEC, and recent enacted legislation has expanded the SEC’s power to seek disgorgement in all FCPA cases filed in federal court and extended the statute of limitations in SEC enforcement actions in intent-based claims such as those under the FCPA from five years to ten years.
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United States Healthcare Reform
The United States and many foreign jurisdictions have enacted or proposed legislative and regulatory changes affecting the healthcare system, including implementing cost-containment programs to limit the growth of government-paid healthcare costs, including price controls, restrictions on reimbursement and requirements for substitution of generic products for branded prescription drugs.
In the United States, the Patient Protection and Affordable Care Act, as amended by the Health Care and Education Affordability Reconciliation Act, or collectively the Affordable Care Act, was intended to broaden access to health insurance, reduce or constrain the growth of healthcare spending, enhance remedies against fraud and abuse, add transparency requirements for the healthcare and health insurance industries, impose new taxes and fees on the health industry and impose additional health policy reforms.
There have been significant ongoing judicial, administrative, executive and legislative efforts to modify or eliminate the Affordable Care Act. Changes to and under the Affordable Care Act remain possible but it is unknown what form any such changes or any law proposed to replace or revise the Affordable Care Act would take, and how or whether it may affect our business in the future. We expect that changes to the Affordable Care Act, the Medicare and Medicaid programs, changes allowing the federal government to directly negotiate drug prices and changes stemming from other healthcare reform measures, especially with regard to healthcare access, financing or other legislation in individual states, could have a material adverse effect on the healthcare industry. In May 2025, an executive order regarding the concept of most-favored nation pricing was issued. Under this order, the Department of Health and Human Services, in coordination with other federal agencies, is directed to take actions to ensure that the price of prescription drugs paid by federal health insurers, including Medicare and Medicaid, is in line with the prices paid in comparably developed nations. We also expect that the Affordable Care Act, as well as other healthcare reform measures that have and may be adopted in the future, may result in more rigorous coverage criteria and in additional downward pressure on the price that we receive for our products and product candidates, if approved. Any reduction in reimbursement from Medicare, Medicaid, or other government programs may result in a similar reduction in payments from private payers.
As an alternative to the Affordable Care Act, the Great Healthcare Plan was recently announced. As presented, the plan is intended to lower drug prices by increasing competition and benchmarking U.S. drug prices to other countries, reduce insurance premiums by redirecting subsidies from insurers to individuals, increase accountability and transparency from insurers, and promote consumer choice by giving individuals more direct control over how healthcare dollars are spent. Legislative and regulatory action will be required to fully implement the plan. It is unclear how these proposed changes will impact our business and the pharmaceutical industry in general.
The Inflation Reduction Act of 2022 (the "IRA") contains substantial drug pricing reforms, including the establishment of a drug price negotiation program within the U.S. Department of Health and Human Services that would require manufacturers to charge a negotiated "maximum fair price" for certain selected drugs or pay an excise tax for noncompliance, the establishment of rebate payment requirements on manufacturers of certain drugs payable under Medicare Parts B and D to penalize price increases that outpace inflation, and requires manufacturers to provide discounts on Part D drugs. Substantial penalties can be assessed for noncompliance with the drug pricing provisions in the IRA. Orphan drugs that treat only one rare disease are exempt from the IRA's drug negotiation program. The IRA could have the effect of reducing the prices we can charge and reimbursement we receive for future products or product candidates, if approved, thereby reducing our profitability, and could have a material adverse effect on our financial condition, results of operations, and growth prospects. The full impact of the IRA on the pharmaceutical industry in general remains uncertain.
At the state level, legislatures have increasingly passed legislation and implemented regulations designed to control pharmaceutical product pricing, including price or patient reimbursement constraints, discounts, restrictions on certain product access and marketing cost disclosure and transparency measures, and in some cases, designed to encourage importation from other countries and bulk purchasing. Additional reform measures may be adopted in the future.
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Human Capital
At Amicus, one of our founding principles is that we believe in each other to foster teamwork and respect for each individuals' contribution. Our passion for making a difference unites us. Supporting our global employees is an essential part of the core values and culture at Amicus. Tied to these values and culture, we believe our success and our ability to help patients depends on our capability to attract, develop and retain key personnel.
As of December 31, 2025, we had 511 full-time employees. As of December 31, 2025, 58% of our global workforce, 27% of our executive management team and 33% of our Board of Directors were women.
Our Mission to ‘Always Put Patients First’ helps keep our employees engaged with this sense of purpose. We support engagement through communicating frequently and transparently with our employees through a variety of communication methods, including video and written communications, town hall meetings, round tables, employee pulse surveys, and Company intranet, and we acknowledge individual contributions through a number of reward and recognition programs. We believe these engagement efforts keep employees informed about our strategy, culture and purpose and motivated to do their best work.
We believe in a strong compliance culture and provide robust trainings to employees on our code of conduct and the various policies contained therein. As part of our commitment to our employees, these trainings cover our zero-tolerance policy towards the use of child labor, forced labor, or other forms of modern slavery, and periodically refreshing the organization’s understanding of our global anti-bribery and corruption policy.
We support and develop our employees through global development programs that build and strengthen employees’ leadership skills through global leadership development programs, targeted development for high-potential talent, development plans and career paths, tuition reimbursement, and the ability to attend industry conferences and trainings, and patient-mission focused Lunch and Learns. Additionally, we strive to attract and retain the most talented employees in the industry and across the globe by offering competitive compensation and benefits that support their health, financial and emotional well-being. Our compensation philosophy is based on rewarding each employee’s individual contributions and striving to achieve equal pay for equal work regardless of gender, race or ethnicity.
Our Corporate Information
We were incorporated under the laws of the State of Delaware on February 4, 2002. The address of our global headquarters is 47 Hulfish Street, Princeton, NJ 08542 and our telephone number is 609-662-2000. Our website address is www.amicusrx.com. We make available free of charge on our website our annual, quarterly, and current reports, including amendments to such reports, as soon as reasonably practicable after we electronically file such material with, or furnish such material to, the U.S. Securities and Exchange Commission.
Information relating to our corporate governance, including our Code of Business Conduct for Employees, Executive Officers and Directors (the "Code of Conduct"), Corporate Governance Guidelines, and information concerning our senior management team, Board of Directors, including Board Committees and Committee charters, and transactions in our securities by directors and executive officers, is available free of charge on our website at www.amicusrx.com under the "Investors — Corporate Governance" caption and in print to any stockholder upon written request to our Chief Legal Officer at the address set forth on the cover of this Annual Report. Any waivers or material amendments to the Code will be posted promptly on our website.
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ITEM 1A. RISK FACTORS
You should carefully consider these risk factors, together with all of the other information included in this Annual Report on Form 10-K, including our Consolidated Financial Statements and the related notes thereto, before you decide whether to make an investment in our securities. The risks set forth below are not the only risks we face. Additional risks and uncertainties not currently known to us or that we currently deem to be immaterial also may materially and adversely affect our business, prospects, financial condition, cash flows, liquidity, funds from operations, results of operations, stock price and ability to service our indebtedness. In such case, the value of our common stock and the trading price of our securities could decline, and you may lose all or a significant part of your investment. Some statements in the following risk factors constitute forward looking statements. Please refer to the explanation of the qualifications and limitations on forward-looking statements under “Forward-Looking Statements” of this Form 10-K.
Risks Related to the Pending Transaction with BioMarin
We may not complete the pending transaction with BioMarin within the time frame we anticipate or at all, which could have an adverse effect on our business, financial results and/or operations.
On December 19, 2025, we entered into the Merger Agreement with BioMarin and its wholly owned acquisition subsidiary. Under the terms of the Merger Agreement and in accordance with the DGCL, if the Merger is completed, BioMarin's wholly owned acquisition subsidiary will merge with and into Amicus, and Amicus will continue as the surviving corporation as a wholly owned subsidiary of BioMarin. In addition, all shares of our common stock outstanding immediately prior to closing (other than Excluded Shares or Dissenting Shares (as defined in the Merger Agreement)) will be cancelled and converted into the right to receive $14.50 per share in cash, without interest and subject to any applicable withholding of taxes. Following completion of the Merger, shares of our common stock will no longer be publicly listed.
The completion of the pending transaction with BioMarin is conditioned upon (i) the adoption of the Merger Agreement by the holders of at least a majority of the outstanding shares of our common stock, (ii) the receipt of certain regulatory approvals, including expiration or termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 and the receipt of any clearance, consent or affirmative approval applicable to the transaction under antitrust laws and foreign direct investment laws of any non-U.S. or supranational governmental bodies listed in the confidential disclosure schedule delivered by Amicus to BioMarin in connection with the execution of the Merger Agreement, including for certain European countries and the Japanese competition authority, (iii) the absence of certain legal restraints preventing or otherwise making illegal the consummation of the Merger, (iv) the accuracy of certain representations and warranties that we made and compliance by us with certain covenants contained in the Merger Agreement, subject to qualifications, (v) there not having been a "Company Material Adverse Effect" (as defined in the Merger Agreement) with respect to us since the date of the Merger Agreement, and (vi) other customary conditions.
In addition, the Merger Agreement may be terminated under certain specified circumstances, including, but not limited to, a change in the recommendation of our Board of Directors or a termination of the Merger Agreement by us to enter into an agreement for a "Superior Offer," as defined in the Merger Agreement. We could also be required to pay BioMarin a termination fee of $175 million if the Merger Agreement is terminated under specific circumstances described in the Merger Agreement. As a result, we cannot assure you that the transaction with BioMarin will be completed, or that, if completed, it will be exactly on the terms set forth in the Merger Agreement or within the expected time frame. If the transaction is not completed within the expected time frame or at all, we may be subject to a number of material risks. The price of our common stock may decline to the extent that current market prices of our common stock reflect a market assumption that the transaction will be completed. The failure to complete the transaction also may result in negative publicity and negatively affect our relationship with our stockholders, employees, collaborators, customers, regulators and other business partners. We may also be required to devote significant time and resources to litigation related to any failure to complete the Merger or related to any enforcement proceeding commenced against us to perform our obligations under the Merger Agreement.
The pendency of the transaction with BioMarin could adversely affect our business, financial results and/or operations.
Our efforts to complete the transaction with BioMarin could cause substantial disruptions in, and create uncertainty surrounding, our business, which may materially adversely affect our results of operation and our business. Uncertainty as to whether the transaction will be completed may affect our ability to recruit prospective employees or to retain and motivate
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existing employees. Employee retention may be particularly challenging while the transaction is pending because employees may experience uncertainty about their roles following consummation of the transaction. A substantial amount of our management's and employees' attention is being directed toward the completion of the transaction and thus is being diverted from our day-to-day operations. Uncertainty as to our future could adversely affect our business and our relationship with vendors, manufacturers, customers, regulators and other business partners. Changes to or termination of existing business relationships could adversely affect our results of operations and financial condition, as well as the market price of our common stock. The adverse effects of the pendency of the transaction could be exacerbated by any delays in completion of the transaction or termination of the Merger Agreement.
While the Merger Agreement is in effect, we are subject to restrictions on our business activities.
While the Merger Agreement is in effect, we are subject to restrictions on our business activities, generally requiring us to conduct our business in the ordinary course and subjecting us to a variety of specified limitations absent BioMarin's prior consent. These limitations include, among other things, restrictions on our ability to incur additional indebtedness, issue additional shares of our common stock outside of existing equity plans, repurchase shares, declare or pay dividends, acquire or dispose of assets or businesses (subject to limited exceptions), enter into or amend certain contracts and make capital expenditures above specified thresholds. These restrictions could prevent us from pursuing strategic business opportunities, taking actions with respect to our business that we may consider advantageous and responding effectively and/or timely to competitive pressures and industry developments, and may as a result materially and adversely affect our business, results of operations and financial condition.
In certain instances, the Merger Agreement requires us to pay a termination fee to BioMarin, which could require us to use available cash that would have otherwise been available for general corporate purposes.
Under the terms of the Merger Agreement, we may be required to pay BioMarin a termination fee of $175 million if the Merger Agreement is terminated under specific circumstances described in the Merger Agreement. If the Merger Agreement is terminated under such circumstances, the termination fee we may be required to pay under the Merger Agreement may require us to use available cash that would have otherwise been available for general corporate purposes and other uses. For these and other reasons, termination of the Merger Agreement could materially and adversely affect our business operations and financial condition, which in turn would materially and adversely affect the price of our common stock.
We have incurred, and will continue to incur, direct and indirect costs as a result of the pending transaction with BioMarin.
We have incurred, and will continue to incur, significant costs and expenses, including fees for professional services and other transaction costs, in connection with the pending transaction. We must pay substantially all of these costs and expenses whether or not the transaction is completed. There are a number of factors beyond our control that could affect the total amount or the timing of these costs and expenses.
Additional lawsuits may be filed against us and the members of our Board of Directors arising out of the Merger, which may delay or prevent the Merger.
Additional putative stockholder complaints, including stockholder class action complaints, and other complaints may be filed against us, our Board of Directors, BioMarin, BioMarin's board of directors and others in connection with the transactions contemplated by the Merger Agreement. The outcome of litigation is inherently uncertain, and we may not be successful in defending against any such future claims. Additional lawsuits that may be filed against us, our Board of Directors, BioMarin, or BioMarin's board of directors could delay or prevent the Merger, divert the attention of our management and employees from our day-to-day business and otherwise adversely affect us financially.
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Risks Related to our Ability to Generate and Sustain Revenue
We depend heavily on sales of Galafold® and increasingly on sales of Pombiliti® + Opfolda®, in Europe, the U.S., Japan, and other geographies. If we are unable to commercialize Galafold® and Pombiliti® + Opfolda® successfully, or experience significant delays in doing so, our business could be materially harmed.
We have invested a significant portion of our efforts and financial resources in the development of Galafold® for the treatment of Fabry disease and in Pombiliti® + Opfolda® for the treatment of Late Onset Pompe Disease and rely upon sales of Galafold® and Pombiliti® + Opfolda® primarily in Europe and the U.S., and growing sales in other geographies. Our ability to generate material product revenues will depend heavily on the successful development, regulatory approval, and commercialization of Galafold® and Pombiliti® + Opfolda® (collectively, "Our Commercial Products"). We will continue to study Our Commercial Products in Phase 4 studies. If the results of the Phase 4 studies negatively change the benefit/risk profile of Our Commercial Products, the commercial success of Our Commercial Products may be substantially diminished. Any adverse market event with respect to Our Commercial Products, including failure to obtain and maintain sufficient market acceptance, could have a material adverse effect on our business, financial condition and results of operations. If our sales of Our Commercial Products were to decrease, or such sales were substantially or completely displaced in the market, or if we are unable to achieve and maintain sufficient market acceptance of Our Commercial Products by physicians, patients, third-party payors and others in the medical community, or if we fail to receive commercial approval in any additional jurisdictions, it could have a material adverse effect on our business, financial condition and results of operations. In addition, if Our Commercial Products were to become the subject of litigation and/or an adverse governmental action requiring us to cease sales of Our Commercial Products, such an event could have a material adverse effect on our business, financial condition and results of operations. In addition, the entry into the market of competitors with new or generic treatments, including oral, ERT and gene therapies, may erode the market for Our Commercial Products and have a material impact on our business.
Any delay or impediment in our ability to obtain regulatory approval in any region to commercialize, or, when approved, obtain coverage and adequate reimbursement from third parties, including government payors, for Our Commercial Products may cause us to be unable to meet our revenue guidance or to generate the revenues necessary to fund our future growth.
Further, the success of Our Commercial Products will depend on a number of factors, including the following:
•obtaining a sufficiently broad label in each territory that would not unduly restrict patient access;
•obtaining additional foreign approvals for Our Commercial Products;
•continuing to build and maintain an infrastructure capable of supporting product sales, marketing, and distribution of Our Commercial Products in the U.S., Europe, Japan and other territories where we pursue commercialization directly;
•maintaining commercial manufacturing arrangements with third-party manufacturers;
•maintaining commercial distribution agreements with third-party distributors;
•launching commercial sales of Our Commercial Products, where approved, whether alone or in collaboration with others;
•obtaining acceptance of Our Commercial Products, where approved, by patients, the medical community, and third-party payors;
•competing effectively with other therapies, including competitor products, potential generics and gene therapies;
•identifying new patients who could benefit from Our Commercial Products successfully;
•continuing an acceptable safety profile of Our Commercial Products;
•obtaining and maintaining patent and trade secret protection and regulatory exclusivity;
•protecting and enforcing our rights in our intellectual property portfolio; and
•obtaining and maintaining a commercially viable price.
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If we do not achieve one or more of these factors in a timely manner or at all, we could experience significant delays or an inability to successfully commercialize Our Commercial Products, which would materially harm our business and ability to meet our financial goals and debt covenants.
If we are not able to obtain, or if there are delays in obtaining, required regulatory approvals, we will not be able to commercialize our products or product candidates and our ability to generate revenue will be materially impaired.
Our commercial products, Galafold® and Pombiliti® + Opfolda®, any product candidates and the activities associated with their development and commercialization, including their testing, manufacture, safety, efficacy, recordkeeping, labeling, storage, approval, advertising, promotion, sale, distribution, commercialization and reimbursement are subject to comprehensive regulation by the European Medicines Agency (“EMA”), the Pharmaceutical and Medical Devices Agency (“PMDA”), the Food and Drug Administration (“FDA”), and other regulatory agencies in the U.S. and by comparable authorities in other countries. Failure to obtain regulatory approval for our products and product candidates, including DMX-200, will prevent us from commercializing our products in jurisdictions beyond those in which we have obtained regulatory approval for our product or in any jurisdictions for our product candidates.
Securing marketing approval for all our product candidates, requires the submission of extensive preclinical and clinical data and supporting information to regulatory authorities for each therapeutic indication to establish the product candidate's safety and efficacy. We will continue to rely on third parties to assist us with filing and supporting the applications necessary to obtain marketing approvals for product candidates in this process. Securing marketing approval also requires the submission of information about the product manufacturing process to, and inspection of manufacturing facilities by, the regulatory authorities. Regulatory authorities may determine that any of our products or product candidates are not effective or only moderately effective, or have undesirable or unintended side effects, toxicities, safety profiles or other characteristics that preclude us from obtaining marketing approval or that prevent or limit their commercial use.
Obtaining approval for all of our product candidates, whether those currently in our pipeline, such as DMX-200, or those we acquire or in-license in the future, is highly uncertain and we may fail to obtain regulatory approval in any or all applicable jurisdictions. The review processes and the processes of regulatory authorities, including the FDA, EMA and PMDA, are extensive, lengthy, expensive, and uncertain, and such regulatory authorities may delay, limit, or deny the approval of any of our product candidates for many reasons, including, but not limited to:
•our failure to demonstrate to the satisfaction of the applicable regulatory authorities that any of our product candidates, are safe and effective for a particular indication;
•the results of clinical trials may not meet the level of statistical significance or other efficacy or safety parameters required by the applicable regulatory authorities for approval;
•the applicable regulatory authority may disagree with the number, design, size, conduct, or implementation of our clinical trials or conclude that the data fail to meet statistical or clinical significance;
•the applicable regulatory authority may not find the data from preclinical studies and clinical trials sufficient to demonstrate that the product candidate's clinical and other benefits outweigh its safety risks;
•the applicable regulatory authority may disagree with our interpretation of data from preclinical studies or clinical trials, and may reject conclusions from preclinical studies or clinical trials, determine that primary or secondary endpoints from clinical trials were not met, or reject safety conclusions from such studies or trials;
•the applicable regulatory authority may not accept data generated at one or more of our clinical trial sites;
•the applicable regulatory authority may determine that we did not properly oversee our clinical trials or follow the regulatory authority's advice or recommendations in designing and conducting our clinical trials;
•an advisory committee, if convened by the applicable regulatory authority, may recommend against approval of our application or may recommend that the applicable regulatory authority require, as a condition of approval, additional preclinical studies or clinical trials, limitations on approved labeling or distribution and use restrictions, or even if an advisory committee, if convened, makes a favorable recommendation, the respective regulatory authority may still not approve the product candidate;
•the applicable regulatory authority may only approve a limited label for less than the full indicated population, as a second line or rescue therapy, or impose other label restrictions; and
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•the applicable regulatory authority may identify deficiencies in the chemistry, manufacturing, and control sections of our application, our manufacturing processes, facilities, or analytical methods or those of our third-party contract manufacturers or be unable to complete any necessary manufacturing inspections of our third-party manufacturers which may lead to significant delays in the approval of our product candidates or to the rejection of our applications altogether.
The process of obtaining marketing approvals is expensive, may take many years, if approval is obtained at all, and can vary substantially based upon a variety of factors, including the type, complexity, and novelty of the product candidates involved. Changes in marketing approval policies during the development period, changes in or the enactment of additional statutes or regulations, or changes in regulatory review for each submitted product application, may cause delays in the approval or rejection of an application. Regulatory authorities have substantial discretion in the approval process and may refuse to accept any application or may decide that our data are insufficient for approval and require additional preclinical, clinical, or other studies. In addition, varying interpretations of the data obtained from preclinical and clinical testing could delay, limit, or prevent marketing approval of a product candidate. Any marketing approval we ultimately obtain may be limited or subject to restrictions or post-approval commitments that render the approved product not commercially viable.
If we are unable to establish and maintain sales and marketing capabilities or enter into agreements with third parties to market and sell our products or, if approved, our product candidates, we may not be successful in commercializing Galafold®, Pombiliti® + Opfolda®, or any product candidate.
To achieve commercial success for any approved product, we must continue to develop and maintain a sales and marketing organization or outsource commercialization to third parties. We have established our own sales and marketing capabilities to promote Galafold® in Europe, Japan, the U.S., and other foreign jurisdictions with a targeted sales force and have leveraged these resources to support the ongoing commercial launch of Pombiliti® + Opfolda® in those same jurisdictions. We anticipate using these capabilities to support other product candidates if approved. We have also entered into distribution agreements with third parties to market our products in jurisdictions in which we do not have our own sales and marketing capabilities. There are risks involved with establishing and maintaining our own sales and marketing capabilities and entering into arrangements with third parties to perform these services for our products or any of our product candidates, if approved. For example, recruiting and training a sales force is expensive and time-consuming and could delay any product launch. If the commercial launch of a product candidate, if approved, for which we recruit a sales force and establish marketing capabilities is delayed or does not occur for any reason, we would have prematurely or unnecessarily incurred these commercialization expenses. This may be costly, and our investment would be lost if we cannot retain or reposition our sales and marketing personnel. Similarly, if we enter into agreements with third parties, including the out licensing of our products or product candidates, we may choose to reduce or eliminate our sales and marketing operations and thereby lose our commercialization investment.
Factors that may inhibit our efforts to successfully commercialize Galafold®, Pombiliti® + Opfolda®, or our product candidates if and when they are approved by regulatory authorities, on our own include:
•our inability to recruit, train, and retain adequate numbers of effective sales and marketing personnel;
•the inability of sales personnel to obtain access to adequate numbers of physicians to prescribe our products;
•the lack of complementary products to be offered by sales personnel, which may put us at a competitive disadvantage relative to companies with more extensive product lines;
•unforeseen costs and expenses associated with creating an independent sales and marketing organization;
•misconduct by independent sales and marketing organizations that expose us to fines, penalties and other restrictions on our ability to effectively market and sell our products; and
•efforts by our competitors to commercialize products at or about the time when our product candidates would be coming to market.
We may also co-promote or out-license our products or product candidates, if approved, in various markets with pharmaceutical and biotechnology companies in instances where we believe that a larger sales and marketing presence will expand the market or accelerate penetration into the market. If we do enter into co-promote or out-licensing arrangements with third parties, our product revenues will be lower than if we directly sold and marketed our products and any revenues received under such arrangements will depend on the skills and efforts of others.
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We may not be successful in entering into distribution arrangements and marketing alliances with third parties. Our failure to enter into these arrangements on favorable terms could delay or impair our ability to commercialize our products and product candidates, if approved, and could increase our costs of commercialization. Dependence on distribution arrangements and marketing alliances to commercialize our products and product candidates will subject us to a number of risks, including:
•we may not be able to control the amount and timing of resources that our distributors may devote to the commercialization of our products or product candidates, if approved;
•our distributors may experience financial difficulties;
•our distributors may experience compliance related issues and associated government investigations;
•our distributors may require transfer of the marketing authorization for our products and product candidates, if approved, and may refuse to relinquish them at the end of the distribution relationship;
•our distributors may be out of compliance with applicable anti-bribery and corruption laws with an adverse effect on operations and expose us to liability;
•business combinations or significant changes in a distributor's business strategy may also adversely affect a distributor's willingness or ability to complete its obligations under any arrangement; and
•these arrangements are often terminated or allowed to expire, which could interrupt the marketing and sales of a product and decrease our revenue.
If we are unable to establish and maintain adequate sales, marketing and distribution capabilities, whether independently or with third parties, we may not be able to generate product revenue at our current guidance, meet our debt obligations, and may not ever become profitable.
If the market opportunities for our products or product candidates are smaller than we believe they are, then our revenues may be adversely affected, and our business may suffer.
Each of the diseases that our products and product candidates are being developed to address is rare and by definition has small patient populations. Our projections of both the number of people who have these diseases, as well as the subset of people with these diseases who have the potential to benefit from treatment with our products and product candidates, are based on estimates.
Currently, most reported estimates of the prevalence of these diseases are based on studies of small subsets of the population of specific geographic areas, which are then extrapolated to estimate the prevalence of the diseases in the broader world population. In addition, as new studies are performed the estimated prevalence of these diseases may change. There can be no assurance that the prevalence of Fabry disease, Pompe disease, or other rare diseases in the study populations, particularly in these newer studies, accurately reflects the prevalence of these diseases in the broader world population. If our estimates of the prevalence of Fabry disease, Pompe disease, or other rare diseases or of the number of patients who may benefit from treatment with our products or product candidates prove to be incorrect, or if we are unable to obtain traditional approval for Galafold® which was granted approval in the U.S. under the accelerated approval program and/or expand the patient populations and approved indications of Galafold® and Pombiliti® + Opfolda® in the jurisdictions in which they are approved, the market opportunities for our products and product candidates, if approved, may be smaller than we believe and our prospects for generating revenue at our guidance levels may be adversely affected and our business may suffer.
Galafold® and Pombiliti® + Opfolda®, or any of our product candidates, including DMX-200, that receive regulatory approval, may fail to achieve the degree of market acceptance by physicians, patients, third-party payors and others in the medical community necessary for commercial success.
Galafold® and Pombiliti® + Opfolda®, as well as any of our product candidates, including DMX-200, that receive regulatory approval may nonetheless fail to gain sufficient market acceptance by physicians, patients, third-party payors and others in the medical community. If these products do not achieve an adequate level of acceptance, we may not generate significant product revenues or any profits from operations. The degree of market acceptance of our product candidates, if approved for commercial sale, will depend on a number of factors, including:
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•the efficacy and potential advantages compared to competitive or alternative treatments, including generics and gene therapies;
•the prevalence and severity of any side effects;
•the ability to offer our products and product candidates, if approved, for sale at competitive prices;
•convenience and ease of administration compared to alternative treatments;
•the willingness of the target patient population to try new therapies and of physicians to prescribe these therapies;
•the strength of marketing and distribution support and timing of market introduction of competitive products;
•publicity concerning our products or competing products and treatments;
•competition from other products for the same or similar indications; and
•sufficient third-party coverage or reimbursement.
Our ability to negotiate, secure and maintain third-party coverage and reimbursement may be affected by political, economic and regulatory developments in the U.S., E.U., U.K. and other jurisdictions. Governments continue to impose cost containment measures, and third-party payors are increasingly challenging prices charged for medicines and examining their cost effectiveness, in addition to their safety and efficacy. These and other similar developments could significantly limit the degree of market acceptance of Galafold®, Pombiliti® + Opfolda® and any of our product candidates that receive marketing approval, and we may fail to meet our revenue targets as a result.
We face substantial competition, which may result in others discovering, developing or commercializing products before or more successfully than we do.
The development and commercialization of new drug products is highly competitive. We face competition with respect to our current products, Galafold®, Pombiliti® + Opfolda® and product candidates, including DMX-200, and will likely face competition for any products we may seek to develop or commercialize in the future, from major pharmaceutical companies, specialty pharmaceutical companies, and biotechnology and gene therapy companies worldwide. For example, several large pharmaceutical and biotechnology companies currently market and sell products for the treatment of lysosomal storage disorders, including Fabry disease and Pompe disease. These Fabry products include Sanofi Aventis' Fabrazyme®, Takeda’s Replagal®, and Chiesi's Elfabrio, as well as other Fabry treatment products in development. As of 2022, Galafold® also faces potential generic competition, with Hatch-Waxman litigation currently on-going. In addition, Sanofi markets and sells Myozyme®, Lumizyme®, Nexviazyme®, Nexviadyme® for the treatment of Pompe disease. We are also aware of other enzyme replacement and substrate reduction therapies in development by third parties for Fabry and Pompe, as well as potential gene therapies for both Fabry and Pompe and our other product candidates.
Potential competitors also include academic institutions, government agencies and other public and private research organizations that conduct research, seek patent protection and establish collaborative arrangements for research, development, manufacturing and commercialization. Our competitors may develop products that are more effective, safer, more convenient or less costly than any that we are developing or that would render our product candidates obsolete or non-competitive. Our competitors may also obtain FDA, EMA, or other regulatory approval for their products more rapidly than we may obtain approval for ours, could achieve regulatory exclusivity and block us from approval and marketing our products for a significant period of time. We may also face competition from off-label use of other approved therapies. There can be no assurance that developments by others will not render our product candidates or any acquired products obsolete or noncompetitive either during the research phase or once the products reach commercialization.
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Many of our competitors have significantly greater financial resources and expertise in research and development, manufacturing, preclinical testing, conducting clinical trials, obtaining regulatory approvals, prosecuting intellectual property rights and marketing approved products than we do. Smaller and other early-stage companies may also prove to be significant competitors, particularly through collaborative arrangements with large and established companies. These third parties compete with us in recruiting and retaining qualified scientific and management personnel, establishing clinical trial sites and patient registration for clinical trials, as well as in acquiring technologies complementary to or necessary for our programs or advantageous to our business. In addition, if we obtain regulatory approvals for our product candidates, manufacturing efficiency and marketing capabilities are likely to be significant competitive factors. We currently rely on third-party manufacturers for all of our products and product candidates. Further, many of our competitors have substantial resources and expertise in conducting collaborative arrangements, sourcing in-licensing arrangements, manufacturing and acquiring new business lines or businesses that are greater than our own.
A variety of risks associated with international operations could materially adversely affect our business.
Galafold®, Pombiliti® + Opfolda®, and any of our product candidates that may be approved in the future for commercialization in the E.U., U.K. or other foreign countries are or will be subject to additional risks related to international operations or entering into international business relationships, including:
•different regulatory requirements for maintaining approval of drugs in foreign countries;
•reduced protection for contractual and intellectual property rights in some countries;
•unexpected changes in taxes, tariffs, trade barriers and regulatory requirements, particularly in light of a new presidential administration in the U.S.;
•economic weakness, including rising interest rates, inflation and political instability in particular foreign economies and markets;
•compliance with tax, employment, immigration and labor laws for employees living or traveling abroad;
•our ability, and our commercialization partners ability, to comply with local laws, rules and regulations, including those relating to modern slavery;
•foreign currency fluctuations, which could result in increased operating expenses and reduced revenue, and other obligations incident to doing business in another country;
•workforce uncertainty in countries where labor unrest is more common than in the U.S.;
•noncompliance with the U.S. Foreign Corrupt Practices Act, the U.K. Bribery Act 2010, and similar anti-bribery and anti-corruption laws in other jurisdictions;
•tighter restrictions on privacy and the collection and use of patient data; and
•business interruptions resulting from geopolitical actions (including war and terrorism), pandemic diseases, or natural disasters (including earthquakes, typhoons, floods and fires).
Moreover, there are complex regulatory, tax, labor, environmental and other legal requirements imposed by the E.U., U.K., and many of the individual countries in Europe, Asia, and Latin America with which we will need to comply. Many U.S.-based biopharmaceutical companies have found the process of marketing their own products in Europe and other international geographies to be very challenging.
In addition, Pombiliti® is currently manufactured in the People's Republic of China (“PRC”) by WuXi Biologics Co., Ltd. (“WuXi”). The PRC, and WuXi specifically, has faced increased scrutiny by the U.S. government, which could impact our ability to supply Pombiliti® to meet our forecasted future demand, as WuXi is our primary supplier. This risk is discussed in greater detail below under the heading “Risks Related to the Manufacture of our Products and Product Candidates and our Dependence on Third Parties”.
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Following the receipt of marketing approval of our products or any product candidates, the products may become subject to unfavorable pricing regulations, third-party coverage, reimbursement practices or healthcare reform initiatives, which would harm our business.
The regulations and practices that govern marketing approvals, pricing, commercialization, coverage and reimbursement for new drug products vary widely from country to country and product to product. Current and future legislation may significantly change the approval requirements in ways that could involve additional costs and cause delays in obtaining approvals. Some countries, including almost all of the member states of the European Economic Area, require approval of the sale price of a drug before it can be marketed. In many countries, the pricing review period begins after marketing or product licensing approval is granted. In some foreign markets, including the European market, prescription pharmaceutical pricing remains subject to continuing governmental control even after initial approval is granted and approved products are subject to re-reviews, class reviews and other governmental controls which can negatively impact pricing originally approved. As a result, we might obtain marketing approval for a product in a particular country, but then be subject to price regulations that delay our commercial launch of the product, possibly for lengthy time periods, and negatively impact any revenues we are able to generate from the sale of the product in that country. Adverse pricing limitations may hinder our ability to recoup our investment in one or more product candidates, even if our product candidates obtain marketing approval.
Our ability to successfully commercialize Galafold®, Pombiliti® + Opfolda®, or any product candidate, including DMX-200, if approved, will also depend in part on the extent to which coverage and reimbursement for these products and related treatments will be available from government health administration authorities, private health insurers and other organizations. Government authorities and other third-party payors, such as private health insurers and health maintenance organizations, decide which medications they will pay for and establish reimbursement levels. A primary trend in the European and U.S. healthcare industries and elsewhere is cost containment. It is currently unknown what impact, if any proposed changes by the federal and state governments in the U.S. and similar changes in foreign countries may have on pricing and reimbursement, particularly with respect to government programs such as Medicare and Medicaid and Pharmacy Benefit Managers for commercial plans, and including reimportation or parallel trade, reference pricing and limitations on manufacturer price increases.
Prices at which we or our customers seek reimbursement for our products can be subject to challenge, reduction or denial by the government and other payers. Increasingly, third-party payors are requiring that drug companies provide them with predetermined discounts from list prices and are challenging the prices charged for pharmaceutical products. We cannot be sure that coverage and reimbursement will continue to be available for Galafold®, Pombiliti® + Opfolda®, or any product candidate that we commercialize or may commercialize, and if coverage and reimbursement are available, what the level of reimbursement will be. Reimbursement may impact the demand for, or the price of, Galafold®, Pombiliti® + Opfolda®, or any product candidate, including DMX-200, for which we obtain marketing approval. Obtaining reimbursement for our product candidates when approved may be particularly difficult because of the higher prices typically associated with drugs directed at smaller populations of patients and the pricing and reimbursement of competitive products. In addition, third-party payors are likely to impose strict requirements for reimbursement of a higher priced drug. If reimbursement is not available or is available only to limited levels, we may not be able to successfully commercialize any product for which we obtain marketing approval.
The U.S. and many foreign jurisdictions have enacted or proposed legislative and regulatory changes affecting the healthcare system, including implementing cost-containment programs to limit the growth of government-paid healthcare costs, including price controls, restrictions on reimbursement and requirements for substitution of generic products for branded prescription drugs. In the U.S., the Affordable Care Act was intended to broaden access to health insurance, reduce or constrain the growth of healthcare spending, enhance remedies against fraud and abuse, add transparency requirements for the healthcare and health insurance industries, impose new taxes and fees on the health industry and impose additional health policy reforms.
There have been significant ongoing judicial, administrative, executive, and legislative efforts to modify or eliminate the Affordable Care Act.
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Changes to and under the Affordable Care Act remain possible but it is unknown what form any such changes or any law proposed to replace or revise the Affordable Care Act would take, and how or whether it may affect our business in the future. We expect that changes to the Affordable Care Act, the Medicare and Medicaid programs, changes allowing the federal government to directly negotiate drug prices and changes stemming from other healthcare reform measures, especially with regard to healthcare access, financing or other legislation in individual states, could have a material adverse effect on the healthcare industry. In May 2025, an executive order was issued implementing the concept of most-favored nation pricing. Under this order, the Department of Health and Human Services, in coordination with other federal agencies, is directed to take actions to ensure that the price of prescription drugs paid by federal health insurers, including Medicare and Medicaid, is in line with the prices paid in comparably developed nations. We also expect that the Affordable Care Act, as well as other healthcare reform measures that have and may be adopted in the future, may result in more rigorous coverage criteria and additional downward pressure on the prices that we receive for our products and product candidates, if approved. Any reduction in reimbursement from Medicare, Medicaid, or other government programs may result in a similar reduction in payments from private payers.
As an alternative to the Affordable Care Act, the Great Healthcare Plan was recently announced. As presented, the plan is intended to lower drug prices by increasing competition and benchmarking U.S. drug prices to other countries, reduce insurance premiums by redirecting subsidies from insurers to individuals, increase accountability and transparency from insurers, and promote consumer choice by giving individuals more direct control over how healthcare dollars are spent. Legislative and regulatory action will be required to fully implement the plan. It is unclear how these proposed changes will impact our business and the pharmaceutical industry in general.
The Inflation Reduction Act of 2022 (the "IRA") contains substantial drug pricing reforms, including the establishment of a drug price negotiation program within the U.S. Department of Health and Human Services that would require manufacturers to charge a negotiated "maximum fair price" for certain selected drugs or pay an excise tax for noncompliance, the establishment of rebate payment requirements on manufacturers of certain drugs payable under Medicare Parts B and D to penalize price increases that outpace inflation, and requires manufacturers to provide discounts on Medicare Part D drugs. Substantial penalties can be assessed for noncompliance with the drug pricing provisions in the IRA. Although the IRA exempts orphan drugs that treat only one rare disease from the drug pricing negotiation provisions, we do not know if additional drug pricing reforms could eliminate this exemption and therefore affect the prices we can charge and reimbursement we receive for our products and product candidates, if approved, thereby reducing our profitability. Any change to the exemption could have a material adverse effect on our financial condition, results of operations, and growth prospects. The full impact of the IRA on the pharmaceutical industry in general remains uncertain.
At the state level, legislatures have increasingly passed legislation and implemented regulations designed to control pharmaceutical product pricing, including price or patient reimbursement constraints, discounts, restrictions on certain product access and marketing cost disclosure and transparency measures, and in some cases, designed to encourage importation from other countries and bulk purchasing. Additional reform measures may be adopted in the future.
The FDA and other regulatory agencies actively enforce the laws and regulations prohibiting the promotion of off-label uses. If we are found to have promoted off-label uses, we may become subject to significant liability.
The FDA and other regulatory agencies strictly regulate the promotional claims that may be made about prescription drug products. In particular, a product may not be promoted in the U.S. for uses that are not approved by the FDA as reflected in the product's approved labeling or prior to regulatory approval. Further, any labeling approved by the FDA for Galafold®, Pombiliti® + Opfolda®,or any of our product candidates may include restrictions on use, limit use to specific populations or include various other limitations. The FDA may impose further requirements or restrictions on the distribution or use of any of our product candidates, if approved, as part of a Risk Evaluation and Mitigation Strategies ("REMS") plan. Physicians may nevertheless prescribe such products to their patients in a manner that is inconsistent with the approved label provided the company did not promote such use. If we are found to have promoted such off-label uses, we may become subject to significant liability. Similarly, the FDA strictly regulates the promotion of investigational products prior to approval, known as pre-approval promotion. The federal government has levied large civil and criminal fines and / or other penalties against companies for alleged improper promotion and has investigated and / or prosecuted several companies in relation to off-label and/or pre-approval promotion. The FDA has also requested that certain companies enter into consent decrees or permanent injunctions under which specified promotional conduct is changed, curtailed, or prohibited or have delayed approval of investigational products due to pre-approval conduct. Inappropriate promotional activities may also subject a company to investigations, prosecutions and litigation by other government entities or private citizens.
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Product liability lawsuits against us could cause us to incur substantial liabilities and limit commercialization of any products that we may develop.
We face an inherent risk of product liability exposure related to the use of our products and product candidates, including risks which may arise from misuse or malfunction of, or design flaws in, such products or product candidates, whether or not such problems directly relate to the products, product candidates and services we have provided. If we cannot successfully defend ourselves against claims that our product candidates or products caused injuries, we will incur substantial and potentially crippling liabilities. Regardless of merit or eventual outcome, liability claims may result in:
•reduced resources of our management to pursue our business strategy;
•decreased demand for any product candidates or products that we may develop;
•injury to our reputation and significant negative media attention;
•regulatory investigations, prosecutions, or enforcement actions that could require costly recalls or product modifications;
•withdrawal of clinical trial participants;
•regulatory authorities placing ongoing clinical trials on clinical hold;
•significant costs to defend the related litigation;
•increased insurance costs or an inability to maintain appropriate insurance coverage;
•substantial monetary awards to trial participants or patients, including awards that substantially exceed our product liability insurance, which we would then be required to pay from other sources, if available, and would damage our ability to obtain liability insurance at reasonable costs, or at all, in the future;
•loss of revenue; and
•the inability to commercialize any products that we may develop.
The amount of insurance that we currently hold may not be adequate to cover all liabilities that we may incur. Insurance coverage is increasingly expensive and we may not be able to maintain insurance coverage at a reasonable cost or in an amount adequate to satisfy any liability that may arise. On occasion, large judgments have been awarded in lawsuits based on drugs that had unanticipated side effects. A successful product liability claim or a series of claims brought against us could cause our stock price to fall and, if judgments exceed our insurance coverage, could decrease our available cash and adversely affect our business including our ability to service our debt and comply with the liquidity and revenue covenants contained therein.
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If the FDA or other applicable regulatory authorities approve generic or biosimilar products that compete with our products or any of our product candidates, it could reduce sales of our products or product candidates.
In the U.S., after an NDA is approved, the product covered thereby becomes a "listed drug," which can, in turn, be cited by potential competitors in support of approval of an abbreviated NDA ("ANDA"). The Federal Food, Drug, and Cosmetic Act ("the FD&C Act"), FDA regulations and other applicable regulations and policies provide incentives to manufacturers to create modified, non-infringing versions of a drug to facilitate the approval of an ANDA or other application for generic substitutes. These manufacturers might only be required to conduct a relatively inexpensive study to show that their product has the same active ingredients, dosage form, strength, route of administration, and conditions of use, or product labeling, as one of our products or product candidates and that the generic product is absorbed in the body at the same rate and to the same extent as, or is bioequivalent to, our product or product candidate. These generic equivalents would be significantly less costly than ours to bring to market and companies that produce generic equivalents are generally able to offer their products at lower prices because they do not need to conduct highly expensive clinical trials to support their ANDA filing. Thus, after the introduction of a generic competitor, a significant percentage of the sales of any branded product are typically lost to the generic product. Accordingly, competition from generic equivalents to our products or product candidates would substantially limit our ability to generate revenues or achieve profitability with a negative impact on continued operations. At the end of 2022, we received Paragraph 4 certifications from three ANDA filers for Galafold® and initiated Hatch-Waxman litigation against these ANDA filers. In the fourth quarter of 2024, we settled the litigation with one of the ANDA filers, and in the fourth quarter of 2025 we settled the litigation with the remaining filers. While we strongly believe in our patent protection and will continue to vigorously defend our Galafold® intellectual property rights, there is no guarantee we will be similarly successful against other challenges to our intellectual property, either in the U.S. or abroad.
The Biologics Price Competition and Innovation Act ("BPCIA") authorizes the FDA to approve "abbreviated" BLAs for products whose sponsors demonstrate they are "biosimilar" to reference products previously approved under BLAs, to which Pombiliti® is subject. The FDA may also separately determine whether "biosimilar" products are "interchangeable" with their reference products. However, the FDA may not approve an "abbreviated" BLA for a biosimilar product until at least twelve years after the date on which the BLA for the reference product was approved. FDA approval of abbreviated BLAs could be further delayed if the reference products are subject to unexpired and otherwise valid patents.
Accordingly, other manufacturers potentially could develop and seek FDA approval of "biosimilar" products at some point in the future, including a biosimilar of Pombiliti® or any other product we develop or acquire that is approved under a BLA.
Our competitors may be able to develop and commercialize their products, including products identical to ours, in any non-U.S. jurisdiction in which we are unable to obtain, maintain, or enforce our patent claims. Furthermore, generic manufacturers may develop, seek approval for and launch generic versions of our products, and may challenge the scope, validity or enforceability of our patents, requiring us to possibly engage in complex, lengthy and costly litigation or other proceedings.
We may expend our limited resources to pursue a particular product, product candidate, or indication and fail to capitalize on a product, product candidates, or indications that may be more profitable or for which there is a greater likelihood of success.
Because we have limited financial and managerial resources, we focus on research programs and product candidates for specific indications. As a result, we may forego or delay pursuit of opportunities with other product candidates or for other indications that later prove to have greater commercial potential. Our resource allocation decisions may cause us to fail to capitalize on viable commercial products or profitable market opportunities. Our spending on current and future research and development programs and product candidates for specific indications may not yield any commercially viable products.
As a result of pursuing the development and commercialization of our product and product candidates using our proprietary and licensed technologies, we may fail to develop other products or product candidates, or address indications based on other scientific approaches that may offer greater commercial potential or for which there is a greater likelihood of success.
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Risks Related to our Products and the Regulatory Approval and Clinical Development of our Product Candidates
Our products or product candidates may cause undesirable side effects or have other properties that could delay or prevent their regulatory approval or commercialization.
Undesirable side effects caused by our products, Galafold® and Pombiliti® + Opfolda®, or product candidates, including DMX-200, could interrupt, delay or halt clinical trials and could result in the withdrawal or denial of regulatory approval by the FDA, EMA or other regulatory authorities for any or all targeted indications, and in turn prevent us from commercializing our products or product candidates, if approved, and generating revenues from their sale. In addition, if we or others identify undesirable side effects caused by our products or product candidates after receipt of marketing approval:
•regulatory authorities may require the addition of restrictive labeling statements;
•regulatory authorities may withdraw their approval of the product; and
•we may be required to change the way the product is administered, or additional clinical trials are conducted.
Any of these events could prevent us from achieving or maintaining market acceptance of the affected product or product candidate or could substantially increase the costs and expenses of commercializing the product or product candidate, if approved, which in turn could delay or prevent us from generating significant revenues from its sale and limiting our ability to meet our financial guidance, debt covenants or adversely affect our reputation.
Any product or product candidate for which we obtain marketing approval could be subject to restrictions or withdrawal from the market and we may be subject to penalties or other enforcement actions if we fail to comply with regulatory requirements or if we experience unanticipated problems with our products or our product candidates, if approved.
Any product or product candidate for which we obtain marketing approval, along with the manufacturing processes, post-approval clinical data, labeling, advertising and promotional activities for such product, will be subject to continual requirements of and review by the FDA, EMA, PMDA and other regulatory authorities. For example, the FDA's requirements include submissions of safety and other post-marketing information and reports, registration requirements, Current Good Manufacturing Practices ("cGMP"), requirements relating to manufacturing, quality control, quality assurance and complaints and corresponding maintenance of records and documents, requirements regarding the distribution of samples to healthcare professionals and recordkeeping. Even if marketing approval of a product candidate is granted, the approval may be subject to limitations on the indicated uses for which the product may be marketed or may be subject to significant conditions of approval, including the requirement of a REMS. The FDA also may impose requirements for costly post-marketing studies or clinical trials and surveillance to monitor the safety or efficacy of the product. For example, Galafold® was approved in the U.S. under the accelerated approval program and is subject to post-marketing requirements to obtain full approval. The labeling, advertising, promotion, marketing and distribution of a drug, biologic, or gene therapy product also must be in compliance with FDA requirements which include, among others, promotional activities, standards and regulations for direct-to-consumer advertising, promotional activities involving the internet and social media platforms, and industry sponsored scientific and educational activities. In general, all product promotion must be consistent with the labeling approved by the FDA for such product, contain a balanced presentation of information on the product's uses, benefits, risks, and important safety information and limitations on use, and otherwise not be false or misleading. The FDA has very broad enforcement authority, and failure to abide by these regulations can result in penalties, including withdrawal of approval, the issuance of a warning letter directing a company to correct deviations from regulatory standards and enforcement actions that can include seizures, injunctions and criminal prosecution. Failure to comply with applicable FDA requirements and restrictions also may subject a company to adverse publicity and enforcement action by the FDA, the U.S. Department of Justice ("DOJ"), the Office of the Inspector General of the U.S. Department of Health and Human Services ("HHS"), or state authorities. This could subject us to a range of penalties that could have a significant commercial impact, including civil and criminal fines and agreements that materially restrict the manner in which a company promotes or distributes its products.
In addition, later discovery of previously unknown adverse events or other problems with our products, manufacturers or manufacturing processes, or failure to comply with regulatory requirements, may yield various results, including:
•restrictions on such products, manufacturers, or manufacturing processes;
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•changes to or restrictions on the labeling or marketing of a product;
•restrictions on product distribution or use;
•requirements to implement a REMS;
•requirements to conduct post-marketing studies or clinical trials;
•warning letters, untitled letters, or Form 483s;
•withdrawal of the products from the market;
•refusal to approve pending applications or supplements to approved applications that we submit;
•recall of products;
•fines, restitution, or disgorgement of profits or revenues;
•suspension or withdrawal of marketing approvals;
•refusal to permit the import or export of our products;
•product seizure;
•injunctions; or
•the imposition of civil or criminal penalties.
Non-compliance with E.U. and U.K. requirements regarding safety monitoring or pharmacovigilance, and with requirements related to the development of products for the pediatric population, can also result in significant financial penalties. Similarly, failure to comply with the E.U.'s and U.K.'s requirements regarding the protection of personal information can also lead to significant penalties and sanctions and business restrictions.
If we, our suppliers, third-party contractors, clinical investigators, or collaborators are slow to adapt, or are unable to adapt, to changes in existing regulatory requirements or adoption of new regulatory requirements or policies, we or our collaborators may lose marketing approval for our products when and if approved, resulting in decreased revenue from milestones, product sales, or royalties.
Our relationships with customers, healthcare providers, patients, patient organizations, charitable foundations, and third-party payors are subject to applicable anti-kickback, fraud and abuse, anti-bribery and corruption and other healthcare laws and regulations, which could expose us to criminal sanctions, civil penalties, contractual damages, reputational harm, and diminished profits and future earnings.
Healthcare providers, physicians, and payors play a primary role in the recommendation and prescription of our products and any product candidates for which we may obtain marketing approval. Increasingly, patients, patient organizations and charitable foundations also can influence selection of and payment for therapies. Our current and future arrangements with payors, healthcare providers, patient organizations, charitable foundations, and patients may expose us to broadly applicable fraud and abuse, anti-bribery and corruption, and other healthcare laws and regulations that may constrain the business or financial arrangements and relationships through which we market, sell, and distribute our products and any product candidates for which we may obtain marketing approval. Even though we do not and will not control referrals of healthcare services or bill directly to Medicare, Medicaid, or other third-party payors, federal, state, and foreign healthcare laws and regulations pertaining to fraud and abuse, anti-bribery and corruption, interaction with patient organizations, charitable foundations, and patients' rights are and will be applicable to our business. Restrictions under applicable federal, state, and foreign healthcare laws and regulations may affect our ability to operate and expose us to areas of risk, including:
•U.S. federal Anti-Kickback Statute, which prohibits, among other things, knowingly and willfully soliciting, offering, receiving or providing remuneration, directly or indirectly, in cash or in kind, to induce or reward either the referral of an individual for, or the purchase, order or recommendation of, any good or service, for which payment may be made under federal and state healthcare programs such as Medicare and Medicaid. A person or entity does not need to have actual knowledge of the statute or specific intent to violate it in order to have committed a violation. Several other countries, including the U.K., have enacted similar anti-kickback, fraud and abuse, and healthcare laws and regulations;
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•U.S. federal False Claims Act, which imposes criminal and civil penalties, including through civil whistleblower or qui tam actions, against individuals or entities for knowingly presenting, or causing to be presented, to the federal government, claims for payment that are false or fraudulent or making a false statement to avoid, decrease or conceal an obligation to pay money to the federal government. In addition, the government may assert that a claim including items and services resulting from a violation of the federal Anti-Kickback Statute constitutes a false or fraudulent claim for purposes of the False Claims Act. In addition, charitable contributions to foundations for use in supporting patients may expose those foundations and us to additional penalties and prosecution under the False Claims Act. There is also a separate false claims provision imposing criminal penalties. Moreover, the Office of Inspector General (“OIG”) issues guidance documents and Advisory Opinions on matters that could give rise to prosecutions, investigations, litigation and/or settlements under the False Claims Act. For example, OIG issued an Advisory Opinion in April 2022 regarding manufacturer support of genetic testing which could form a basis for government scrutiny in certain circumstances. Applicable regulations of both the EMA and E.U. member states also impose liability for failing to comply with fraud and abuse laws or improperly using information obtained in in the course of clinical trials with the EMA or other regulatory authorities;
•U.S. federal Health Insurance Portability and Accountability Act of 1996 ("HIPAA") which imposes criminal liability and amends provisions on the reporting, investigation, enforcement, and penalizing of civil liability for executing a scheme to defraud any healthcare benefit program or making false statements relating to healthcare matters. Similar to the federal Anti-Kickback Statute, a person or entity does not need to have actual knowledge of the statute to defraud any healthcare benefit program or specific intent to violate it in order to have committed a violation. This statute also may impose monetary penalties on any offers or transfers of remuneration to Medicare or Medicaid beneficiaries (patients) which is likely to influence the beneficiary's selection of particular supplier of government payable items. States, such as California have enacted their own privacy regulations and others may enact similar legislation. Similarly, the collection and use of personal health data in the E.U. is governed by the E.U. General Data Protection Regulation (the "GDPR"), with many requirements mandated by the GDPR for the consent of the individuals to whom the personal data relates, the information provided to the individuals, transfer of personal data within and outside of the E.U., and the security and confidentiality of the personal data. Failure to comply with the requirements of the GDPR may result in substantial fines and other administrative penalties. The GDPR increases our responsibility and liability in relation to personal data that we process, and we may be required to put in place additional mechanisms ensuring compliance with the GDPR. This may be onerous and adversely affect our business, financial condition, results of operations, and prospects;
•HIPAA, as amended by the Health Information Technology for Economic and Clinical Health Act of 2009, and its implementing regulations, which also imposes obligations on certain covered entity healthcare providers, health plans, and healthcare clearinghouses as well as their business associates that perform certain services involving the use or disclosure of individually identifiable health information, including mandatory contractual terms, with respect to safeguarding the privacy, security and transmission of individually identifiable health information;
•U.S. federal laws requiring drug manufacturers to report annually information related to certain payments and other transfers of value made to physicians (defined to include doctors, dentists, optometrists, podiatrists chiropractors, physician assistants, nurse practitioners, clinical nurse specialists, certified nurse anesthetists, and certified nurse-midwives) and teaching hospitals, as well as ownership or investment interests held by physicians and their immediate family members, including under the federal Open Payments program, commonly known as the Sunshine Act, as well as other state and foreign laws regulating marketing activities and requiring manufacturers to report marketing expenditures, payments and other transfers of value to physicians and other healthcare providers. Similarly, payments made to physicians in certain E.U. member states must be publicly disclosed. Moreover, agreements with physicians often must be the subject of prior notification and approval by the physician's employer, his or her competent professional organization and/or the regulatory authorities of the individual E.U. member states. These requirements are provided in the national laws, industry codes or professional codes of conduct, applicable in the E.U. member states. In addition, the provision of benefits or advantages to physicians to induce or encourage the prescription, recommendation, endorsement, purchase, supply, order or use of medicinal products is prohibited in the E.U. Failure to comply with these requirements could result in reputational risk, public reprimands, administrative penalties, fines, or imprisonment;
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•U.S. federal government price reporting laws, which require us to calculate and report complex pricing metrics to government programs, where such reported prices may be used in the calculation of reimbursement and/or discounts on our marketed drugs. Participation in these programs and compliance with the applicable requirements may subject us to potentially significant discounts on our products, increased infrastructure costs, potential liability for the failure to report such prices in an accurate and timely manner, and potentially limit our ability to offer certain marketplace discounts;
•U.S. Foreign Corrupt Practices Act (the "FCPA"), which prohibits us and third parties working on our behalf from making payments to foreign government officials to assist in obtaining or retaining business. Specifically, the anti-bribery provisions of the FCPA prohibit the willful use of the mail or any means of instrumentality of interstate commerce corruptly in furtherance of any offer, payment, promise to pay, or authorization of the payment of money or anything of value to any person, while knowing that all or a portion of such money or thing of value will be offered, given or promised, directly or indirectly, to a foreign official to influence the foreign official in his or her official capacity, induce the foreign official to do or omit to do an act in violation of his or her lawful duty, or to secure any improper advantage in order to assist in obtaining or retaining business for or with, or directing business to, any person; enforcement actions may be brought by the Department of Justice or the SEC; legislation has expanded the SEC’s power to seek disgorgement in all FCPA cases filed in federal court and extended the statute of limitations in SEC enforcement actions in intent-based claims, such as those under the FCPA, from five years to ten years; and
•state and foreign equivalents of each of the above laws, including foreign anti-bribery and corruption laws and state anti-kickback and false claims laws, which may apply to sales or marketing arrangements and claims involving healthcare items or services reimbursed by non-governmental payors, including private insurers; state laws which require pharmaceutical companies to comply with the pharmaceutical industry's voluntary compliance guidelines and the relevant compliance guidance promulgated by the federal government or otherwise restricting payments that may be made to healthcare providers; and state and foreign laws governing the privacy and security of health information in certain circumstances, many of which differ from each other in significant ways and often are not preempted by HIPAA, thus complicating compliance efforts.
While we do not submit claims and our customers will make the ultimate decision on how to submit claims, in the U.S. we may provide reimbursement guidance and support regarding Galafold® or Pombiliti® + Opfolda®, as well as our product candidates for which we receive regulatory approval, to our customers and patients. If a government authority were to conclude that we provided improper advice to our customers and patients and/or encouraged the submission of false claims for reimbursement, we could face action by government authorities. Similarly, if a government authority were to conclude that our patient support efforts or interactions with charitable foundations were improper, we could face action by government authorities. While we have processes and controls to ensure that our business arrangements with third parties will comply with applicable healthcare laws and regulations, it is nonetheless possible that governmental authorities will conclude that our business practices may not comply with current or future statutes, regulations, or case law involving applicable fraud and abuse or other healthcare laws and regulations. If our operations are found to be in violation of any of these laws or any other governmental regulations that may apply to us, we may be subject to significant civil, criminal, and administrative penalties, damages, fines, imprisonment, exclusion from participation in government funded healthcare programs, such as Medicare and Medicaid, and the curtailment or restructuring of our operations.
If clinical trials of our product candidates fail to demonstrate safety and efficacy to the satisfaction of the FDA, EMA, PMDA, or other foreign regulatory authorities, or do not otherwise produce favorable results, we may experience delays in completing, or ultimately be unable to complete, the development and commercialization of our product candidates.
In connection with seeking marketing approval from regulatory authorities for the sale of any product candidate, we must complete preclinical development and then conduct extensive clinical trials to demonstrate the safety and efficacy of our product candidates in humans. Clinical testing is expensive, difficult to design and implement, can take many years to complete, and is uncertain as to outcome. A failure of one or more clinical trials can occur at any stage of testing. The outcome of preclinical testing and early clinical trials may not be predictive of the success of later clinical trials, and interim results of a clinical trial do not necessarily predict final results. Moreover, preclinical and clinical data are often susceptible to varying interpretations and analyses, and many companies that have believed their product candidates performed satisfactorily in preclinical studies and clinical trials have nonetheless failed to obtain marketing approval of their products. If the results of these trials or tests are not positive or are only modestly positive; or if there are safety concerns, we may:
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•choose not to seek regulatory approval in the U.S., E.U., U.K., or other key jurisdictions;
•be delayed in obtaining marketing approval for our product candidates;
•not obtain marketing approval at all;
•obtain approval for indications or patient populations that are not as broad as intended or desired;
•obtain approval with labeling that includes significant use or distribution restrictions or safety warnings, including boxed warnings;
•be subject to additional post-marketing testing requirements, safety strategies or restrictions, such as a requirement of a risk evaluation and mitigation strategy, or REMS; or
•have the product removed from the market after obtaining regulatory approval.
In addition, we do not know whether any preclinical tests or clinical trials will begin as planned, need to be restructured, or be completed on schedule or at all. Significant preclinical study or clinical trial delays could also shorten any periods during which we may have the exclusive right to commercialize our product candidates, if approved, allow our competitors to bring products to market before we do, or impair our ability to successfully commercialize our product candidates, which may harm our business and results of operations.
If we experience any of a number of possible unforeseen events in connection with our clinical trials, potential regulatory approval or commercialization of our product candidates, if approved, could be delayed or prevented.
We may experience numerous unforeseen events during, or as a result of, clinical trials that could delay or prevent our ability to receive regulatory approval or commercialize our product candidates, including:
•clinical trials of our product candidates may produce negative or inconclusive results, and we may decide, or regulators may require us, to conduct additional clinical trials or abandon product development programs;
•the number of patients required for clinical trials of our product candidates may be larger than we anticipate, enrollment in these clinical trials may be slower than we anticipate, or patients may drop out of these clinical trials at a higher rate than we anticipate;
•we may be unable to enroll a sufficient number of patients in our trials to ensure adequate statistical power to detect any statistically significant treatment effects;
•our third-party contractors may fail to comply with regulatory requirements or meet their contractual obligations to us in a timely manner, or at all;
•regulators, institutional review boards, or independent ethics committees may not authorize us or our investigators to commence a clinical trial or conduct a clinical trial at a prospective trial site;
•we may have delays in reaching or fail to reach agreement on acceptable clinical trial contracts or clinical trial protocols with prospective trial sites;
•we may have to suspend or terminate clinical trials of our product candidates for various reasons, including a finding that the participants are being exposed to unacceptable health risks;
•regulators, institutional review boards, or independent ethics committees may require that we or our investigators suspend or terminate clinical research for various reasons, including noncompliance with regulatory requirements or a finding that the participants are being exposed to unacceptable health risks;
•the cost of clinical trials of our product candidates may be greater than we anticipate;
•the supply or quality of our product candidates or other materials necessary to conduct clinical trials of our product candidates may be insufficient or inadequate;
•our product candidates may have undesirable side effects or other unexpected characteristics, causing us or our investigators, regulators, institutional review boards or independent ethics committees to suspend or terminate the trials; or
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•we may in-license product candidates and, by the terms of such licenses, need to rely on third parties to conduct certain clinical trials that may be designed, powered or otherwise conducted in a manner over which we may not have absolute control.
Our product development costs will increase if we experience delays in testing or regulatory approvals. We do not know whether any preclinical tests or clinical trials will begin as planned, will need to be restructured or will be completed on schedule, or at all. Significant preclinical study or clinical trial delays also could shorten any periods during which we may have the exclusive right to commercialize our product candidates, allow our competitors to bring products to market before we do, or impair our ability to successfully commercialize our product candidates, and so may harm our business and results of operations.
If we experience delays or difficulties in the enrollment of patients in our clinical trials, our receipt of necessary regulatory approvals could be delayed or prevented.
We may not be able to initiate or continue clinical trials for our product candidates if we are unable to locate and enroll a sufficient number of eligible patients to participate in these trials. The diseases that our product candidates, Galafold® and Pombiliti® + Opfolda® are intended to treat are characterized by small patient populations, which could result in slow enrollment of clinical trial participants. In addition, our competitors have ongoing clinical trials for product candidates that could be competitive with our product candidates. As a result, potential clinical trial sites may elect to dedicate their limited resources to participation in our competitors' clinical trials and not ours, and patients who would otherwise be eligible for our clinical trials may instead enroll in clinical trials of our competitors' product candidates.
Patient enrollment is affected by other factors including:
•severity of the disease under investigation;
•eligibility criteria for the clinical trial in question;
•perceived risks and benefits of the product candidate under study;
•efforts to facilitate timely enrollment in clinical trials;
•patient referral practices of physicians;
•the ability to monitor patients adequately during and after treatment; and
•proximity and availability of clinical trial sites for prospective patients.
Enrollment delays in our clinical trials may result in increased development costs for our product candidates, which would cause the value of the Company to decline and limit our ability to obtain additional financing. Our inability to enroll a sufficient number of patients in any of our clinical trials would result in significant delays or may require us to abandon one or more clinical trials altogether.
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We may not be able to obtain or maintain orphan drug exclusivity for our products or product candidates. If our competitors are able to obtain orphan drug exclusivity for their products, we may not be able to have competing products approved by the applicable regulatory authority for a significant period of time.
Regulatory authorities in some jurisdictions, including the E.U., U.K., and U.S., may designate drugs for relatively small patient populations as orphan drugs. Amicus currently has orphan drug designation from the FDA, E.U. and U.K., Japan, South Korea, Switzerland and Taiwan for Galafold® for the treatment of Fabry disease. Amicus currently has orphan drug designation from the FDA, U.K., Japan and Switzerland for Pombiliti® + Opfolda® for the treatment of Pompe disease. DMX-200 also has orphan drug designation from the FDA for the treatment of FSGS. Our competitors have also received orphan designations. However, these orphan designations may be retracted following regulatory review of our or our competitor’s marketing authorization and/or BLA submissions and may not be reflected in the final approval of a product. Generally, if a product with an orphan drug designation subsequently receives the first marketing approval for the indication for which it has such designation, the product is entitled to a period of market exclusivity, which, subject to certain exceptions, precludes the EMA from approving another marketing application for a similar medicinal product or the FDA from approving another marketing application for the same drug for the same indication for that time period. The FDA defines “same drug” as a drug or biologic that contains the same active moiety and is intended for the same use. The applicable market exclusivity period for orphan drugs is ten years in the E.U. and U.K. and seven years in the U.S. The E.U. and U.K. exclusivity period can be reduced to six years if a drug no longer meets the criteria for orphan drug designation, including if the drug is sufficiently profitable so that market exclusivity is no longer justified.
Even if we obtain orphan drug exclusivity for other product candidates in these indications, we may not be able to maintain it. For example, if a competitive product that is the same drug or a similar medicinal product as our product or product candidate is shown to be clinically superior to our product or product candidate, as applicable, any orphan drug exclusivity we have obtained will not block the approval of such competitive product. In addition, orphan drug exclusivity will not prevent the approval of a product that is the same drug as our product or product candidate if the FDA finds that we cannot assure the availability of sufficient quantities of the drug to meet the needs of the persons with the disease or condition for which the drug was designated.
Failure to obtain or maintain regulatory approval in foreign jurisdictions would prevent us from marketing our products abroad.
In order to market and sell our products in Europe and many other jurisdictions, we must obtain separate marketing approvals and comply with numerous and varying regulatory requirements. The approval procedure varies among countries and can involve additional testing. The time required to obtain approval may differ from that required to obtain FDA approval. The regulatory approval process outside the U.S. generally includes all of the risks associated with obtaining FDA approval. In addition, some countries outside the U.S. require approval of the sales price of a drug before it can be marketed. In many countries, separate procedures must be followed to obtain reimbursement. We may not obtain marketing, pricing or reimbursement approvals outside the U.S. on a timely basis, if at all. Approval by one regulatory authority does not ensure approval by other regulatory authorities. We may not be able to file for marketing approvals and may not receive necessary approvals to commercialize our products in any market. Regulatory approvals in countries outside the U.S. do not ensure pricing approvals in those countries or in any other countries, and regulatory approvals and pricing approvals do not ensure that reimbursement will be obtained. Moreover, our therapy for the treatment of Pompe disease is comprised of two components, an ERT (Pombiliti®) and a small molecule (Opfolda®). Full marketing approval is required for both components in each jurisdiction that we seek to commercialize in and a failure to secure marketing approval for either in a given target geography could materially harm our business and results of operations.
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Risks Related to the Manufacture and Distribution of our Products and Product Candidates and our Dependence on Third Parties
Use of third parties to manufacture our products or product candidates may increase the risk that we will not have sufficient quantities of our products or product candidates or such quantities at an acceptable cost, which could delay, prevent or impair our development or commercialization efforts.
We do not currently own or operate manufacturing facilities for clinical or commercial production of our products or product candidates. We currently lack the resources and the capabilities to manufacture ourselves on a clinical or commercial scale. If we choose in the future to manufacture ourselves, we would face all of the risks and uncertainties of third-party manufacturers of our products. We currently outsource all manufacturing and packaging of our products and product candidates to third parties. The manufacture of pharmaceutical products requires significant expertise and capital investment, including the development of advanced manufacturing techniques and process controls. In particular, the manufacture of our biologic product for Pompe is highly complex and we may encounter difficulties in production. These problems include difficulties with production costs and yields and quality control, including stability of the product or product candidate. The occurrence of any of these problems could significantly delay our clinical trials or the commercial availability of our products or product candidates.
We may be unable to enter into agreements for commercial supply with third-party manufacturers or may be unable to do so on acceptable terms. Even if we enter into these agreements, the manufacturers of each product or product candidate will be single source suppliers to us for a significant period of time.
Even if we are able to establish and maintain arrangements with third-party manufacturers, reliance on third-party manufacturers entails additional risks, including:
•reliance on the third party for regulatory compliance and quality assurance, including with their own vendors with which we do not have a contractual relationship;
•limitations on supply availability resulting from capacity, scheduling constraints, and geographic of the third parties;
•inability to manufacture product that meets the regulatory requirements for product approval;
•inability to manufacture batches that meet specifications and quality standards;
•inability to hire and retain the skilled workers necessary to manufacture our products;
•inability to meet environmental sustainability requirements;
•impact on our reputation in the marketplace if manufacturers of our products, once commercialized, fail to meet the demands of our customers;
•the possible breach of the manufacturing agreement by the third party;
•the possible misappropriation of our proprietary information, including our trade secrets and know-how;
•the high cost of manufacturing processes and raw materials; and
•the possible termination or nonrenewal of the agreement by the third party at a time that is costly or inconvenient for us.
The failure of any of our contract manufacturers to maintain high manufacturing standards could result in injury or death of clinical trial participants or patients using products. Such failure could also result in product liability claims, product recalls, product seizures or withdrawals, delays or failures in testing or delivery, cost overruns or other problems that could seriously harm our business, profitability, or reputation.
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The FDA and regulatory authorities in other jurisdictions require our contract manufacturers to comply with cGMP regulations. These regulations cover all aspects of the manufacturing, testing, quality control and recordkeeping relating to our product candidates and any products that we may commercialize, including Galafold®, Pombiliti® + Opfolda®, and DMX-200, if approved. The FDA and other regulatory authorities may, and often will, require the inspection of our contract manufacturers in order to approve, or maintain the approval of, our products or product candidates, including Galafold® and Pombiliti® + Opfolda®. Different geopolitical situations or other unforeseeable events could impact the FDA, or other regulatory authorities, ability to timely inspect such contract manufacturers and such delays could materially harm our business and accuracy of our financial guidance projections.
Our contract manufacturers may not be able to comply with cGMP regulations or similar regulatory requirements outside the U.S. Our failure or the failure of our third party manufacturers, to comply with applicable regulations could significantly and adversely affect regulatory approval and supplies of our products and product candidates. Our products and product candidates that we may develop may compete with other product candidates and products for access to manufacturing facilities. There are a limited number of manufacturers that operate under cGMP regulations and that might be capable of manufacturing our products and product candidates.
The majority of our product candidates and commercial products, including Galafold® and Pombiliti®, are manufactured by single source third party manufacturers. If the third parties that we engage to manufacture our product candidates and our commercial products should cease to continue to do so for any reason, we likely would experience delays in advancing the development of our product candidates and continuing to commercialize our products while we identify and qualify replacement suppliers and we may be unable to obtain replacement suppliers on terms that are favorable to us or in a timely fashion. In addition, if we are not able to obtain adequate supplies of our product candidates, commercial products, or the drug substances used to manufacture them, it will be more difficult for us to develop our product candidates and continue to commercialize our products and compete effectively.
Our current and anticipated future dependence upon others for the manufacture of our product candidates may adversely affect our future profit margins, our ability to meet our obligations under our credit facility, and our ability to develop product candidates and commercialize any products that receive regulatory approval on a timely and competitive basis.
We rely on third parties to distribute our products, and those third parties may not perform satisfactorily, including failing to deliver products to meet demand.
We do not distribute our products ourselves and rely on third parties for the delivery of clinical and commercial products to our customers and patients. Any of these third parties may experience delays in the delivery of our products, may be unable to deliver the products or may not comply with the appropriate delivery conditions. Failure to deliver our products may adversely affect our future profit margins, our ability to meet our obligations under our credit facility and our ability to develop and commercialize our products.
We rely on third parties to conduct certain preclinical development activities and our clinical trials, and those third parties may not perform satisfactorily, including failing to meet deadlines for the completion of such trials.
We do not independently conduct clinical trials for our product candidates or certain preclinical development activities of our product candidates. We rely on third parties, such as contract research organizations ("CROs"), clinical data management organizations, medical institutions, clinical investigators, and collaboration partners to perform these functions. Any of these third parties may terminate their engagements with us at any time. If we need to enter into alternative arrangements, it would delay our product development activities.
Our reliance on these third parties for certain preclinical and clinical development activities reduces our control over these activities but does not relieve us of our responsibilities. The FDA requires us to comply with standards, commonly referred to as Good Clinical Practices ("GCP"), for conducting, recording and reporting the results of clinical trials to assure that data and reported results are credible and accurate and that the rights, integrity, and confidentiality of trial participants are protected. We also are required to register certain ongoing clinical trials and post the results of completed clinical trials on a government-sponsored database, ClinicalTrials.gov, within particular timeframes. Failure to do so can result in fines, adverse publicity and civil and criminal sanctions. Similar GCP and transparency requirements apply in the E.U. and U.K. Failure to comply with such requirements, including with respect to clinical trials conducted outside the E.U., U.K., and U.S., can also lead regulatory authorities to refuse to take into account clinical trial data submitted as part of an MAA.
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Furthermore, third parties that we rely on for our clinical development activities may also have relationships with other entities, some of which may be our competitors. If these third parties do not successfully carry out their contractual duties, meet expected deadlines or conduct our clinical development activities in accordance with regulatory requirements or our stated protocols, we will not be able to obtain, or may be delayed in obtaining, marketing approvals for our product candidates and will not be able to, or may be delayed in our efforts to, successfully commercialize our product candidates. Our product development costs will increase if we experience delays in testing or obtaining marketing approvals.
We also rely on other third parties to obtain, store, and distribute drug supplies for our preclinical development activities and clinical trials. In addition, in some instances we are required to purchase clinical supplies from our competitors, who may refuse to allow this purchase or do so at prohibitively high prices. Any performance failure on the part of our distributors or inability to secure supply from our competitors could delay preclinical and clinical development or marketing approval of our product candidates or commercialization of our products, producing additional losses and depriving us of potential product revenue.
Extensions, delays, suspensions, or terminations of our preclinical development activities or our clinical trials as a result of the performance of our independent clinical investigators and CROs will delay, and make more costly, regulatory approval for any product candidates that we may develop or acquire. Any change in a CRO during an ongoing preclinical development activity or clinical trial could seriously delay that trial and potentially compromise the results of the activity or trial.
We may not be successful in maintaining or establishing collaborations, which could adversely affect our ability to develop and, particularly in international markets, commercialize products.
We are collaborating with physicians, academic institutions, hospitals, patient advocacy groups, foundations, and government agencies in order to assist with the development of our products and each of our product candidates. We also collaborate closely with Dimerix, a clinical stage biopharmaceutical company who we have licensed the U.S. rights to their product candidate, DMX-200. We plan to pursue similar activities in future programs and plan to evaluate the merits of retaining commercialization rights for ourselves or entering into selective collaboration arrangements with leading pharmaceutical or biotechnology companies. We also may seek to establish collaborations for the sales, marketing, and distribution of our products in all or select geographies. If we elect to seek collaborators in the future but are unable to reach agreements with suitable collaborators, we may fail to meet our business objectives for the affected product or program. We face, and will continue to face, significant competition in seeking appropriate collaborators. Moreover, collaboration arrangements are complex and time consuming to negotiate, document, and implement. We may not be successful in our efforts, if any, to establish and implement collaborations or other alternative arrangements. The terms of any collaboration or other arrangements that we establish, if any, may not be favorable to us.
Any collaboration that we enter into may not be successful. The success of our collaboration arrangements, if any, will depend heavily on the efforts and activities of our collaborators. It is likely that any collaborators of ours will have significant discretion in determining the efforts and resources that they will apply to these collaborations. The risks that we may be subject to in possible future collaborations include the following:
•collaborators have significant discretion in determining the efforts and resources that they will apply to these collaborations;
•collaborators may fail to fulfill their contractual obligations;
•we may be unable to retain collaborators who are qualified to assist in the development and commercialization of our products or product candidates;
•collaborators may not pursue development and commercialization of our products or product candidates or may elect not to continue or renew development or commercialization programs, based on clinical trial results, changes in the collaborators' strategic focus or available funding, or external factors such as an acquisition that diverts resources or creates competing priorities;
•collaborators may delay clinical trials, provide insufficient funding for a clinical trial program, stop a clinical trial or abandon a product candidate, repeat or conduct new clinical trials or require a new formulation of a product candidate for clinical testing;
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•collaborators could independently develop, or develop with third parties, products that compete directly or indirectly with our products or product candidates if the collaborators believe that competitive products are more likely to be successfully developed or can be commercialized under terms that are more economically attractive than ours;
•a collaborator with marketing and distribution rights to one or more products may not commit sufficient resources to the marketing and distribution of such product or products;
•collaborators may not properly maintain or defend our intellectual property rights or may use our proprietary information in such a way as to invite litigation that could jeopardize or invalidate our intellectual property or proprietary information or expose us to potential liability;
•collaborators may infringe the intellectual property rights of third parties, which may expose us to litigation and potential liability;
•disputes may arise between the collaborator and us as to the ownership of intellectual property arising during the collaboration;
•we may grant rights to our collaborators to be the holder of any marketing authorizations in a jurisdiction, which could impact our ability to successfully commercialize our products;
•we may grant exclusive rights to our collaborators, which would prevent us from collaborating with others;
•disputes may arise between the collaborators and us that result in the delay or termination of the research, development or commercialization of our products or product candidates or that result in costly litigation or arbitration that diverts management attention and resources; and
•collaborations may be terminated and, if terminated, may result in a need for additional capital to pursue further development or commercialization of the applicable product candidates.
Collaboration agreements may not lead to development or commercialization of product candidates in the most efficient manner or at all. If a collaborator of ours were to be involved in a business combination, the continued pursuit and emphasis on our product development or commercialization program could be delayed, diminished, or terminated.
Collaborations with pharmaceutical companies and other third parties often are terminated or allowed to expire by the other party. Such terminations or expirations may adversely affect us financially and could harm our business reputation in the event we elect to pursue collaborations that ultimately expire or are terminated.
Materials necessary to manufacture our products or product candidates may not be available on commercially reasonable terms, or at all, which may delay the development and commercialization of our products or product candidates.
We currently rely on the manufacturers of our products and product candidates to purchase from third-party suppliers the materials necessary to produce the compounds for our preclinical studies, clinical trials, and commercial supply and we rely, or will rely, on these or other manufacturers for commercial distribution of our products and, if and when we obtain marketing approval, for any of our product candidates. Suppliers may not sell these materials to our manufacturers at the time we need them or on commercially reasonable terms and all such materials are susceptible to fluctuations in price and availability due to transportation costs, government regulations, price controls, geopolitical risk and changes in economic climate or other foreseen circumstances. We do not have any control over the process or timing of the acquisition of these materials by our manufacturers. We may enter into agreements to purchase certain materials and provide them to our manufacturers, with all the risks and uncertainties of supply associated with those purchases. If we or our manufacturers are unable to obtain these materials for our preclinical studies and clinical trials, product testing and potential regulatory approval of our product candidates would be delayed, significantly impacting our ability to develop and commercialize our product candidates. If our manufacturers or we are unable to purchase these materials for commercial distribution of our approved products, the commercial launch of our products and product candidates would be delayed or there would be a shortage in supply, which would materially affect our ability to generate revenues from the sale of our products or product candidates.
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Manufacturing issues may arise that could increase product and regulatory approval costs or delay commercialization.
Manufacturing of our products and product candidates requires us or our manufacturing partners to conduct required stability and comparability testing. In the future we may identify impurities which could result in increased scrutiny by regulatory authorities, delays in our clinical programs and regulatory approval, increases in our operating expenses, or failure to obtain or maintain approval for our products or product candidates. We or our partners may also encounter product, packaging, equipment and process-related issues that may require refinement or resolution in order to successfully commercialize our products, proceed with planned clinical trials, or obtain regulatory approval for commercial marketing of our product candidates. New environmental laws or regulations in the various jurisdictions in which we operate may also impose additional requirements that impact the way our products are manufactured or packaged. Complying with such changes could be costly, and a failure to comply in a timely manner could lead to fines, penalties, or the inability to commercialize such products in those jurisdictions, materially impacting our revenue, cash flow, and financial guidance targets.
As noted above, we currently rely on WuXi, as the primary supplier of our biologic product, Pombiliti®. Accordingly, there is a risk that supplies of our product may be significantly delayed by, or may become unavailable as a result of, manufacturing, equipment, process, regulatory or business-related issues affecting that company. We may also face additional manufacturing and supply-chain risks due to the regulatory and political structure of the PRC, or as a result of the international relationship between the PRC and the U.S., including but not limited to potential new regulations and/or sanctions imposed by the U.S. government on the PRC or WuXi specifically, such as the BIOSECURE Act which prohibits entities that receive federal funds from using biotechnology that is from a company associated with a foreign adversary, or any of the other countries in which our products are marketed. While WuXi is currently not subject to the BIOSECURE Act, there is no guarantee they will continue to remain exempt. There is additional uncertainty as it is not known what actions, including the imposition of sanctions or tariffs, may be taken by the presidential administration in the U.S. In addition, the out-breaks of illnesses in the PRC could impact operations at WuXi. Although currently there has been no impact on our ability to obtain supply of Pombiliti®., and we and WuXi have robust mitigation plans in place to the extent feasible based on the risk, there can be no assurance that operations would not be impacted in the future with a negative impact on supply of our product.
We are currently working with WuXi to develop a second manufacturing facility for Pombiliti® in Dundalk, Ireland. This facility will require substantial time and investment to ensure it meets applicable regulatory standards governing the manufacture of commercial pharmaceutical products. Although this facility is EMA approved, there can be no assurance that this facility will be approved by the FDA or other similar regulatory bodies. As a result, this facility may experience delays in getting approved or require an additional outlay of capital. There can be no guarantee that we will see any return on investment in the near term or ever.
Risks Related to our Financial Position
We have incurred significant losses since our inception and anticipate that we will continue to incur losses in the future.
To date, we have focused on developing and commercializing our first product, Galafold® and second therapy, Pombiliti® + Opfolda®, as well as our pipeline therapies, including DMX-200 in the U.S. Investment in pharmaceutical product development is highly speculative because it entails substantial upfront capital expenditures and significant risk that a product candidate will fail to gain regulatory approval or become commercially viable. Although the European Commission, PMDA, FDA, and other regulatory authorities have granted approval for Galafold®, for the treatment of adults with a confirmed diagnosis of Fabry disease and who have an amenable genetic variant, as well as Pombiliti® + Opfolda® for the treatment of adults with Pompe disease, and we are generating product sales, we continue to incur significant research, development, commercialization, manufacturing, and other expenses related to our ongoing operations. As a result, we have incurred losses in each period since our inception and may not be profitable on a non-GAAP or GAAP basis or achieve our year-to-year profitability or cash flow guidance.
We expect to continue to incur significant costs in the foreseeable future as we:
•continue our development and commercialization of our products and seek regulatory approvals for our product candidates in the U.S., E.U., U.K., Japan and other foreign countries, as applicable;
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•conduct additional clinical trials to support the full approval of Galafold® in the U.S. and post-approval commitments or trials in the E.U. and other geographies;
•continue communicating with the EMA, as necessary, regarding post-marketing requirements and clinical trials for Galafold®;
•continue to or initiate the regulatory submission process for marketing approval of Galafold® and Pombiliti® + Opfolda® outside of the U.S. and E.U. and other foreign jurisdictions where approved, as applicable;
•build and maintain our commercial infrastructure so that it is capable of supporting product sales, marketing and distribution of Galafold® and Pombiliti® + Opfolda®, as well as our other product candidates in Europe, Japan, the U.S., or other territories in which we have received or may receive regulatory approval;
•continue our next-generation product research;
•prepare for the U.S. commercial launch of DMX-200, should it be approved by the FDA; and
•continue our rigorous prosecution and defense of our patent portfolio.
We may encounter unforeseen expenses, difficulties, complications, delays, and other unknown factors that may adversely affect our business. We may also engage in various types of business development activities which could adversely impact our cash position or profitability projections. The size of our future losses will depend, in part, on the rate of future growth of our expenses and our ability to generate revenues. If any of our product candidates fails in clinical trials or does not gain regulatory approval, or if approved, fails to achieve market acceptance, we may never become fully profitable. Even if we achieve profitability in the future, we may not be able to sustain profitability in subsequent periods. Our prior losses and potential future losses have had and will continue to have an adverse effect on our stockholders' equity and working capital.
We may never become profitable even though we currently generate revenue from the sale of products.
Our ability to generate material revenue and become profitable depends upon our ability to successfully commercialize our existing products and product candidates, or product candidates that we may in-license or acquire in the future. Even if we are able to successfully achieve regulatory approval for our product candidates, we do not know when any of these product candidates will generate revenue for us, if at all and we may not meet our current revenue, operating expense and profitability guidance. Our ability to generate revenue from our current or future products and product candidates depends on a number of factors, including our ability to:
•successfully complete development activities and obtain additional regulatory, pricing, and reimbursement approvals for, and continue to successfully commercialize, Galafold® and Pombiliti® + Opfolda®;
•complete regulatory submissions and obtain regulatory approval in target geographies for Pombiliti® + Opfolda®;
•develop and maintain a commercial organization capable of sales, marketing, and distribution for Galafold®, Pombiliti® + Opfolda®, and any product candidates we intend to market if approved, in the countries where we have chosen to commercialize the products ourselves, including the U.S., E.U., U.K., and Japan;
•manufacture commercial quantities of our products at acceptable cost levels;
•obtain a commercially viable price for our products;
•obtain coverage and adequate reimbursement from third parties, including government payors;
•successfully satisfy post-marketing requirements that the FDA, EMA, or other foreign regulatory authorities may impose for Galafold®, Pombiliti® + Opfolda®, or any of our other product candidates that may receive regulatory approval, including pediatric trials and patient registries;
•successfully develop or acquire new products and product candidates;
•successfully complete development activities, including the necessary pre-clinical studies and clinical trials, with respect to product candidates;
•successfully protect our intellectual property rights; and
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•successfully navigate the evolving geopolitical landscape and any adverse impacts arising therefrom, including actions by governments, our customers, our suppliers or other third parties.
Even if we are able to generate significant revenues from the sale of our products and accurately predict and control expenses, we may not reach our financial guidance or become profitable and may need to obtain additional funding to continue operations. If we fail to become profitable or are unable to sustain profitability on a continuing basis, we may be unable to continue our operations at planned levels and be forced to reduce our operations.
If we require substantial additional capital to fund our operations and fail to obtain necessary financing, we may be unable to complete the development and commercialization of our products and development and commercialization of our product candidates.
Our operations have consumed substantial amounts of cash. We expect to continue to spend substantial amounts to advance the preclinical and clinical development of our product candidates, and launch and commercialize our products and product candidates for which we may receive regulatory approval, including continuing to maintain our own commercial organization. We believe that excluding any impact of the pending consummation of the Merger, our current cash position and expected revenues are sufficient to fund our operations and ongoing research programs for at least the next 12 months. Potential impacts of global pandemics, government sanctions, future business development collaborations, pipeline expansion, and investment in manufacturing capabilities could impact our future capital requirements. As such, we may require substantial additional capital for the development and commercialization of our products and further development and commercialization of our product candidates.
If additional funding is needed, we cannot be certain that such funding will be available on acceptable terms, or at all. If we are unable to raise additional capital in sufficient amounts, when required or on acceptable terms, we could also be required to:
•significantly delay, scale back, or discontinue the development or commercialization of our products or product candidates or one or more of our other research and development initiatives;
•seek collaborators for Galafold®, Pombiliti® + Opfolda®, or one or more of our current or future product candidates at an earlier stage than otherwise would be desirable, or on terms that are less favorable than might otherwise be available;
•relinquish or license on unfavorable terms our rights to our technologies, products or product candidates that we otherwise would seek to develop or commercialize ourselves;
•significantly curtail operations; or
•enter into strategic partnerships on unfavorable terms, including a sale of our assets for less than full value.
Our forecast of the period of time through which our financial resources will be adequate to support our operations is a forward-looking statement and involves risks and uncertainties, and actual results could vary as a result of a number of factors, including the factors discussed elsewhere in this "Risk Factors" section. We have based this estimate on assumptions that may prove to be wrong, and we could utilize our available capital resources sooner than we currently expect. Our future funding requirements, both near and long-term, will depend on many factors, including, but not limited to:
•the costs of commercialization activities, including maintaining sales, marketing, and distribution capabilities for Galafold®, Pombiliti® + Opfolda®, and any product candidates for which we may receive regulatory approval in regions where we choose to commercialize our products on our own, including DMX-200 in the U.S.;
•the scope, progress, results, and costs of preclinical development, laboratory testing, and clinical trials for our product candidates and any other product candidates that we may in-license or acquire;
•the cost of manufacturing drug supply for our preclinical studies, clinical trials, and commercial supply, including the significant cost of manufacturing Pombiliti® + Opfolda® and our gene therapies;
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•the outcome, timing, and cost of the regulatory approval process by the FDA, EMA, PMDA, and other foreign regulatory authorities, including the potential for regulatory authorities to delay approvals pending site inspections or requiring that we perform more studies than those that we currently anticipate for our products and product candidates;
•the outcome and timing of the reimbursement and pricing approvals by the applicable authorities in the jurisdictions we are seeking to commercialize our products;
•the activities of our competitors;
•the cost of filing, prosecuting, defending, and enforcing any patent claims and other intellectual property rights;
•the cost and timing of completion of existing or expanded commercial-scale outsourced manufacturing activities;
•the cost of defending any claims asserted against us or prosecuting any claims we may assert against others;
•the impact of, and cost of complying with new laws, rules, regulations or executive orders in the geographies in which we or our key manufacturers, suppliers and customers operate;
•the emergence of competing technologies and other adverse market developments;
•the impact of foreign exchange rates on our operating expenses and revenue projections; and
•the extent to which we acquire or invest in additional businesses, products, and technologies.
Raising additional capital may cause dilution to our existing stockholders, restrict our operations, or require us to relinquish rights to our technologies, Galafold®, Pombiliti® + Opfolda®, or product candidates.
If the Merger is not consummated, we may seek additional capital through a combination of private and public equity offerings, debt financings, receivables or royalty financings, strategic collaborations and alliances, restructuring and licensing arrangements. We have an effective “shelf” registration statement on Form S-3 that allows us to issue securities in registered offerings as well as an available at-the-market financing facility that allows us to sell shares of our common stock through a placement agent at market prices. To the extent that we raise additional capital through the sale of equity, warrants or convertible debt securities, your ownership interest will be diluted, and the terms may include liquidation or other preferences that adversely affect the rights of existing stockholders. Debt, receivables, and royalty financings may be coupled with an equity component, such as warrants to purchase stock, which could also result in dilution of our existing stockholders' ownership. The incurrence of additional indebtedness beyond our existing indebtedness with the Senior Secured Term Loan due 2029 could also result in increased fixed payment obligations and could also result in certain restrictive covenants, such as limitations on our ability to incur further debt, limitations on our ability to acquire or license intellectual property rights, and other operating restrictions that could have a material adverse effect on our ability to conduct our business and may result in liens being placed on our assets and intellectual property. If we were to default on any of our indebtedness, we could lose such assets and intellectual property. If we raise additional funds through strategic collaborations and alliances and licensing arrangements with third parties, we may have to relinquish valuable rights to Galafold®, Pombiliti® + Opfolda® or our product candidates, or grant licenses on terms that are not favorable to us. If we are unable to raise additional funds through equity or debt financing when needed, we may be required to delay, limit, reduce or terminate our product development or commercialization efforts or grant rights to develop and market our technologies that we would otherwise prefer to develop and market ourselves.
Servicing our debt requires a significant amount of cash, and we may not have sufficient cash flow from our business to pay our substantial debt.
In October 2023, we entered into the Senior Secured Term Loan due 2029 for a $400 million credit facility with Blackstone Alternative Credit Advisors LP and Blackstone Life Sciences Advisors L.L.C. (collectively, “Blackstone”), with an interest rate equal to a 3-month adjusted Term SOFR, subject to a 2.5% floor, plus 6.25% per annum that requires interest-only payments until late-2026 and matures in 2029. We received net proceeds of $387.4 million in October 2023, after deducting fees and expenses. There were no warrants or equity conversion features associated with the Senior Secured Term Loan due 2029, but Blackstone simultaneously made a $29.8 million investment in our common stock, net of offering costs. The Senior Secured Term Loan due 2029 contains a minimum liquidity covenant, tested monthly, and a minimum consolidated revenue covenant measured as of the previous four consecutive fiscal quarters over the term of the loan.
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There can be no assurance that our cash and cash equivalents, together with funds generated by our operations and any future financings, will be sufficient to satisfy our debt payment obligations or that we will have sufficient equity to satisfy these obligations. Our inability to generate sufficient funds to satisfy our debt payment obligations or remain in compliance with the debt covenants may result in such obligations being accelerated by our lenders, which would likely have a material adverse effect on our business, financial condition and results of operations.
Foreign currency exchange rate fluctuations could harm our financial results.
We conduct operations in many countries involving transactions denominated in a variety of currencies other than the U.S. dollar. The majority of our current Galafold® and Pombiliti® + Opfolda® revenue is derived from outside the U.S. Accordingly, changes in the value of currencies relative to the U.S. dollar may impact our consolidated revenues and operating results due to transactional and translational remeasurement that is reflected in our earnings. The current exposures arise primarily from cash, accounts receivable, intercompany receivables and payables, and net product sales denominated in foreign currencies. Fluctuations in currency exchange rates have had, and will continue to have, an impact on our results as expressed in U.S. dollars. We are not currently engaged in any foreign currency hedging activities and there can be no assurance that currency exchange rate fluctuations will not adversely affect our results of operations, financial condition and cash flows. Adverse fluctuations in currency exchange rates from the date of our outlooks could cause our actual results to differ materially from those anticipated in our outlooks and adversely impact our business, results of operations and financial condition.
We also face risks arising from the imposition of exchange controls and currency devaluations. Exchange controls may limit our ability to convert foreign currencies into U.S. dollars or to make payments by our foreign subsidiaries or businesses located in or conducted within a country imposing controls. Currency devaluations result in a diminished value of funds denominated in the currency of the country instituting the devaluation.
Our ability to use our net operating loss carryforwards and certain other tax attributes may be limited.
As of December 31, 2025, we had U.S. federal and state net operating loss carry forwards ("NOLs") of approximately $1.2 billion and $1.0 billion, respectively. The federal carry forward for losses generated prior to 2018 will expire in 2033 through 2037. Federal net operating losses incurred in 2018 and onward have an indefinite expiration under the Tax Act. Most of the state net operating loss carry forwards generated prior to 2009 have expired through 2016. The remaining state net operating loss carry forwards including those generated in 2009 through 2025 will expire in 2028 through 2045. State research and development credits will expire 2026 through 2033. Utilization of NOLs may be subject to a substantial limitation pursuant to Section 382 of the Internal Revenue Code of 1986, as amended ("Section 382"), as well as similar state statutes in the event of an ownership change. Such ownership changes have occurred in the past and could occur again in the future. Under Section 382, if a corporation undergoes an "ownership change," generally defined as a greater than 50% change (by value) in its equity ownership over a three-year period, the corporation's ability to use its pre-change NOLs and other pre-change tax attributes (such as research and development tax credits) to offset its post-change income may be limited. We may experience ownership changes in the future as a result of shifts in our stock ownership some of which are outside our control. We completed a detailed study of the NOLs for the tax year 2024 and determined that there was not an ownership change in excess of 50%. Ownership changes in future periods may place additional limits on our ability to utilize net operating loss and tax credit carry forwards. In addition, at the state level, there may be periods during which the use of NOLs is suspended or otherwise limited, which could accelerate or permanently decrease the amount of state attributes and increase state taxes owed.
Our executive officers, directors and principal stockholders maintain the ability to exert significant influence and control over matters submitted to our stockholders for approval.
Our executive officers, directors and principal stockholders beneficially own shares representing approximately 20% of our common stock as of February 12, 2026. As a result, if these stockholders were to choose to act together, they would be able to exert significant influence and control over matters submitted to our stockholders for approval, as well as our management and affairs. For example, these persons, if they choose to act together, could influence the election of directors and approval of any merger, consolidation, sale of all or substantially all of our assets or other business combination or reorganization. This concentration of voting power could delay or prevent an acquisition of us on terms that other stockholders may desire. The interests of this group of stockholders may not always coincide with the interests of other stockholders, and they may act, whether by meeting or written consent of stockholders, in a manner that advances their best interests and not necessarily those of other stockholders, including obtaining a premium value for their common stock, and might affect the prevailing market price for our common stock.
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Because we do not anticipate paying any cash dividends on our capital in the foreseeable future, capital appreciation, if any, will be our stockholders sole source of gain.
We have never declared or paid cash dividends on our capital stock. We currently intend to retain all of our future earnings, if any, to finance the development and growth of our business. In addition, the Merger Agreement with BioMarin generally restricts, subject to certain limited exceptions, our ability to pay cash dividends on our capital stock during the interim period between the execution of the Merger Agreement and the completion of the transaction (or the date on which the Merger Agreement is earlier terminated) and the terms of any future debt agreements may preclude us from paying dividends. As a result, capital appreciation, if any, of our common stock will be our stockholders sole source of gain for the foreseeable future.
Our disclosure controls and procedures may not prevent or detect all errors or acts of fraud.
We are subject to the periodic reporting requirements of the Securities Exchange Act of 1934, as amended (the "Exchange Act"). Our disclosure controls and procedures are designed to reasonably assure that information required to be disclosed by us in reports we file or submit under the Exchange Act is accumulated and communicated to management, recorded, processed, summarized and reported within the time periods specified in the rules and forms of the SEC. We believe that any disclosure controls and procedures, no matter how well conceived and operated, can provide only reasonable, not absolute, assurance that the objectives of the control system are met.
These inherent limitations reflect the reality that judgments can be faulty, and that breakdowns can occur because of simple error or mistake. Additionally, controls can be circumvented by the individual acts of some persons, by collusion of two or more people or by an unauthorized override of the controls. Accordingly, because of the inherent limitations in our control system, misstatements due to error or fraud may occur and not be detected.
Risks Related to our Intellectual Property
If we are unable to obtain and maintain patent protection for our technology and products, or if the scope of the patent protection is not sufficiently broad, our competitors could develop and commercialize technology and products similar or identical to ours, and our ability to successfully commercialize our technology and products may be adversely affected.
Our success depends in large part on our ability to obtain and maintain patent protection in the U.S. and other countries with respect to our proprietary technology and products. We seek to protect our proprietary position by filing patent applications in the U.S. and in certain foreign jurisdictions related to our novel technologies, products and product candidates that are important to our business. This process is expensive and time-consuming, and we may not be able to file and prosecute all necessary or desirable patent applications at a reasonable cost or in a timely manner. It is also possible that we will fail to identify patentable aspects of our research and development output before it is too late to obtain patent protection. Moreover, if we license technology or product candidates from third parties in the future, these license agreements may not permit us to control the preparation, filing and prosecution of patent applications, or to maintain or enforce the patents, covering this intellectual property. These agreements could also give our licensors the right to enforce the licensed patents without our involvement, or to decide not to enforce the patents at all. Therefore, in these circumstances, these patents and applications may not be prosecuted and enforced in a manner consistent with the best interests of our business.
The patent position of biotechnology and pharmaceutical companies generally is highly uncertain, involves complex legal and factual questions and has in recent years been the subject of much litigation, including U.S. Hatch-Waxman litigation. As a result, the issuance, scope, validity, enforceability and commercial value of our patent rights are highly uncertain. Our pending and future patent applications may not result in patents being issued which protect our technology or products, in whole or in part, or which effectively prevent others from commercializing competitive technologies and products. Changes in either the patent laws or interpretation of the patent laws in the U.S. and other countries may diminish the value of our patents or narrow the scope of our patent protection.
The degree of future protection for our proprietary rights is uncertain, and we cannot ensure that:
•we or our licensors were the first to make the inventions covered by each of our pending patent applications;
•we or our licensors were the first to file patent applications for these inventions;
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•others will not independently develop similar or alternative technologies or duplicate any of our technologies;
•any patents issued to us or our licensors will provide a basis for commercially viable products, will provide us with any competitive advantages or will not be challenged by third parties;
•licenses from other third parties will not be required to commercialize patented products;
•we will develop additional proprietary technologies that are patentable;
•we will file patent applications for new proprietary technologies promptly or at all;
•our patents will not expire prior to or shortly after commencing commercialization of a product;
•the patents of others will not have a negative effect on our ability to do business;
•patent authorities will not identify deficiencies in our patent applications and refuse to grant our patents; or
•outcome of any patent litigation involving Galafold®, or any possible future litigation involving Pombiliti® + Opfolda®, will demonstrate that our patents are valid and enforceable.
In addition, we cannot be assured that any of our pending patent applications will result in issued patents. In particular, we have filed patent applications in the U.S., the European Patent Office and other countries outside the U.S. that have not been issued as patents. These pending applications include, among others, some of the patent applications for Pombiliti® + Opfolda®, and Galafold®. If patents are not issued in respect of our pending patent applications, we may not be able to stop competitors from marketing similar products in Europe and other countries in which we do not have issued patents.
In addition to patent protection outside of the U.S., we intend to seek orphan medicinal product designation of our product candidates and to rely on statutory data exclusivity provisions in jurisdictions outside the U.S. where such protections are available, including Europe. The patent rights that we own or have licensed relating to our product candidates are limited in ways that may affect our ability to exclude third parties from competing against us if we obtain regulatory approval to market these product candidates. We have multiple U.S. composition of matter patents covering Galafold® and multiple method of treatment patents issued and listed in the Orange Book. We have composition of matter, method of treatment, method of manufacture, formulation and other patents issued for Pombiliti® + Opfolda®. We also have several pending applications globally covering Galafold®, Pombiliti® + Opfolda® and our product candidates. There can be no assurance that these applications will be allowed, that allowed applications will be issued, or that the scope of such patents, if they issue, will be sufficient to protect our products. Composition of matter patents can provide protection for pharmaceutical products to the extent that the specifically covered compositions are important. For our product candidates for which we do not hold composition of matter patents, competitors who obtain the requisite regulatory approval can offer products with the same composition as our products so long as the competitors do not infringe any method of use patents that we may hold. This type of patent does not limit a competitor from making and marketing products that are identical to our products that is labeled for an indication that is outside of the patented method, or for which there is a substantial use in commerce outside the patented method.
Additionally, physicians may prescribe such competitive identical products for indications other than the one for which the products have been approved, or off-label indications, that are covered by the applicable patents. Although such off-label prescriptions may infringe or induce infringement of method of use patents, the practice is common and such infringement is difficult to prevent or prosecute.
The laws of foreign countries may not protect our rights to the same extent as the laws of the U.S. For example, European patent law restricts the patentability of methods of treatment of the human body more than U.S. law does. Certain foreign jurisdictions may not recognize or enforce any patents granted or patent applications filed in those jurisdictions. In addition, we may not pursue or obtain patent protection in all major markets. Assuming the other requirements for patentability are met, currently, the first to file a patent application is generally entitled to the patent. However, prior to March 16, 2013, in the U.S., the first to invent was entitled to the patent. Publications of discoveries in the scientific literature often lag behind the actual discoveries, and patent applications in the U.S. and other jurisdictions are typically not published until 18 months after filing, or in some cases not at all. Therefore, we cannot know with certainty whether we were the first to make the inventions claimed in our patents or pending patent applications, or that we were the first to file for patent protection of such inventions.
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Additionally, we may be subject to a third-party pre-issuance submission of prior art to the U.S. Patent and Trademark Office or become involved in opposition, derivation, reexamination, inter partes review, post grant review, interference proceedings or other patent office proceedings or litigation, in the U.S. or elsewhere, challenging our patent rights or the patent rights of others, including U.S. Hatch-Waxman litigation. An adverse determination in any such submission, proceeding or litigation could reduce the scope of, or invalidate, our patent rights, allow third parties to commercialize our technology or products and compete directly with us, without payment to us, or result in our inability to manufacture or commercialize products without infringing third-party patent rights. In addition, if the breadth or strength of protection provided by our patents and patent applications is threatened, it could dissuade companies from collaborating with us to license, develop or commercialize current or future product candidates.
Even if our patent applications issue as patents, they may not issue in a form that will provide us with any meaningful protection, prevent competitors from competing with us or otherwise provide us with any competitive advantage. Our competitors may be able to circumvent our owned or licensed patents by developing similar or alternative technologies or products in a non-infringing manner. In addition, other companies may attempt to circumvent any regulatory data protection or market exclusivity that we obtain under applicable legislation, which may require us to allocate significant resources to preventing such circumvention. Legal and regulatory developments in the E.U. and elsewhere may also result in clinical trial data submitted as part of an MAA becoming publicly available. Such developments could enable other companies to circumvent our intellectual property rights and use our clinical trial data to obtain marketing authorizations in the E.U. and in other jurisdictions. Such developments may also require us to allocate significant resources to prevent other companies from circumventing or violating our intellectual property rights. Our attempts to prevent third parties from circumventing our intellectual property and other rights may ultimately be unsuccessful. We may also fail to take the required actions or pay the necessary fees to maintain our patents.
The issuance of a patent is not conclusive as to its inventorship, scope, validity or enforceability, and our owned and licensed patents may be challenged in the courts or patent offices in the U.S. and abroad. Such challenges may result in loss of exclusivity or freedom to operate or in patent claims being narrowed, invalidated or held unenforceable, in whole or in part, which could limit our ability to stop others from using or commercializing similar or identical technology and products, or limit the duration of the patent protection of our technology and products. Given the amount of time required for the development, testing and regulatory review of new product candidates, patents protecting such candidates might expire before or shortly after such candidates are commercialized. As a result, our patent portfolio may not provide us with sufficient rights to exclude others from commercializing products similar or identical to ours.
Further, litigation, interferences, oppositions, inter partes reviews, administrative challenges or other similar types of proceedings are, have been and may in the future be necessary in some instances to determine the validity and scope of certain of our proprietary rights, and in other instances to determine the validity, scope or non-infringement of certain patent rights claimed by third parties to be pertinent to the manufacture, use or sale of our products. We may also face challenges to our patent and regulatory protections covering our products by third parties, including manufacturers of generics and biosimilars that may choose to launch or attempt to launch their products before the expiration of our patent or regulatory exclusivity. Litigation, interference, oppositions, inter partes reviews, administrative challenges or other similar types of proceedings are unpredictable and may be protracted, expensive and distracting to management. The outcome of such proceedings could adversely affect the validity and scope of our patent or other proprietary rights, hinder our ability to manufacture and market our products, require us to seek a license for the infringed product or technology or result in the assessment of significant monetary damages against us that may exceed amounts, if any, accrued in our financial statements. An adverse determination in a judicial or administrative proceeding or a failure to obtain necessary licenses could prevent us from manufacturing or selling our products and result in significant financial losses if such adverse determination relates to Galafold® in the U.S. E.U., U.K., or other major market from which we derive substantial revenue from an approved product. Furthermore, payments under any licenses that we are able to obtain would reduce our profits derived from the covered products and services.
Additionally, our products, or the technologies or processes used to formulate or manufacture those products may now, or in the future, infringe the patent rights of third parties. It is also possible that third parties will obtain patents or other proprietary rights that might be necessary or useful for the development, manufacture or sale of our products. We may need to obtain licenses for intellectual property rights from others and may not be able to obtain these licenses on commercially reasonable terms, if at all.
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We may become involved in Hatch-Waxman litigation to protect or enforce our patents or other intellectual property, which could be expensive, time consuming, and unsuccessful.
There has been, and we expect that there may continue to be, significant litigation in the industry regarding patents and other intellectual property rights. Litigation, arbitrations, administrative proceedings and other legal actions with private parties and governmental authorities concerning patents and other intellectual property rights may be protracted, expensive and distracting to management. Competitors may sue us as a way of delaying the introduction of our drugs or to remove our drugs from the market. Any litigation, including litigation related to an ANDA, litigation related to 505(b)(2) applications, interference proceedings to determine priority of inventions, derivations proceedings, inter partes review, oppositions to patents in foreign countries, litigation against our collaborators or similar actions, may be costly and time consuming and could harm our business. We expect that litigation may be necessary in some instances to determine the validity and scope of certain of our proprietary rights. Litigation may be necessary in other instances to determine the validity, scope or non-infringement of certain patent rights claimed by third parties to be pertinent to the manufacture, use or sale of our products. Ultimately, the outcome of such litigation could adversely affect the validity and scope of our patent or other proprietary rights, hinder our ability to manufacture and market our products, or result in the assessment of significant monetary damages against us that may exceed amounts, if any, accrued in our financial statements.
To the extent that valid present or future third-party patents or other intellectual property rights cover our drugs, drug candidates or technologies, we or our strategic collaborators may seek licenses or other agreements from the holders of such rights in order to avoid or settle legal claims. Such licenses may not be available on acceptable terms, which may hinder our ability to, or prevent us from being able to, manufacture and market our drugs. Payments under any licenses that we are able to obtain would reduce our profits derived from the covered products.
As part of the approval process for Galafold®, the FDA granted us a New Chemical Entity (“NCE”) exclusivity period during which other manufacturers’ applications for approval of generic versions of our product will not be approved. Generic manufacturers may challenge the patents protecting products that have been granted NCE exclusivity one year prior to the end of the NCE exclusivity period. Generic manufacturers have sought and may continue to seek FDA approval for a similar or identical drug through an ANDA, the application form typically used by manufacturers seeking approval of a generic drug. The sale of generic versions of Galafold® earlier than their patent expiration would have a significant negative effect on our revenues and results of operations. To seek approval for a generic version of a product having NCE status, a generic company may submit its ANDA to the FDA four years after the branded product’s approval.
In October 2022, we received Paragraph IV Notice letters from Aurobindo Pharma Ltd. (along with their applicable affiliates, “Aurobindo”), Lupin Ltd. (along with their applicable affiliates, “Lupin”), and Teva Pharmaceutical, Inc. (along with their applicable affiliates “Teva”, and collectively Teva, Aurobindo and Lupin, the “generic manufacturers”) indicating that they have each submitted ANDAs to the FDA requesting permission to market and manufacture generic versions of Galafold®. They have challenged the validity of all or some of the patents listed in the Orange Book for Galafold®. We filed patent infringement lawsuits in the U.S. District Court for the District of Delaware within forty-five days of receiving each Paragraph IV Notice Letter against each of the generic manufacturers triggering a 30-month stay of FDA regulatory approval of each ANDA.
On October 15, 2024, we entered into a license agreement with Teva to resolve their ANDA litigation. Under the terms of the license, among other things, Teva received a non-exclusive, non-transferable, royalty-free, fully paid-up license to commercialize its generic version of Galafold® in the U.S., commencing on January 30, 2037, or earlier in certain circumstances. The license agreement was submitted to the Federal Trade Commission and the Department of Justice, pursuant to applicable law, for their review. On December 19, 2025, we announced that we have entered into settlement agreements with Aurobindo and Lupin on substantially similar terms as Teva. The agreements with all three parties concluded our Galafold® Hatch-Waxman litigation. Nonetheless, we may face future litigation on our other products or product candidates. The Company intends to continue to vigorously defend its patent rights, both domestically and internationally; however, the outcome of such matters cannot be predicted with certainty.
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Third parties may initiate legal proceedings alleging that we are infringing their intellectual property rights, the outcome of which would be uncertain and could have a material adverse effect on the success of our business.
Our research, development and commercialization activities, as well as any product candidates or products resulting from these activities, including Galafold® or Pombiliti® + Opfolda®, may infringe or be accused of infringing one or more claims of an issued patent or may fall within the scope of one or more claims in a published patent application that may subsequently issue and to which we do not hold a license or other rights. Third parties may own or control these patents or patent applications in the U.S. and abroad. These third parties could bring claims that would cause us to incur substantial expenses and, if successful against us, could cause us to pay substantial damages. Further, if a patent infringement suit were brought against us, we or they could be forced to stop or delay research, development, manufacturing or sales of the products or product candidate that is the subject of the suit.
No assurance can be given that patents do not exist, have not been filed, or could not be filed or issued, which contain claims covering our product candidates, technology or methods. Because of the number of patents issued and patent applications filed in our field, we believe there is a risk that third parties may allege they have patent rights encompassing our product candidates, technology or methods.
If any of these patents were to be asserted against us, while we do not believe that our product candidates would be found to infringe any valid claim of such patents, there is no assurance that a court would find in our favor. If we were to challenge the validity of any issued U.S. patent in court, we would need to overcome a presumption of validity that attaches to every patent. This burden is high and would require us to present clear and convincing evidence as to the invalidity of the patent's claims. Furthermore, during the course of litigation, confidential information may be disclosed in the form of documents or testimony in connection with discovery requests, depositions or trial testimony. Disclosure of our confidential information and our involvement in intellectual property litigation could materially adversely affect our business.
In order to avoid or settle potential claims with respect to any patent rights of third parties, we may choose or be required to seek a license from a third party and be required to pay license fees, royalties, or both. These licenses may not be available on acceptable terms, or at all. Even if we or our collaborators were able to obtain a license, the rights may be nonexclusive, which could result in our competitors gaining access to the same intellectual property. Ultimately, we could be prevented from commercializing a product, or be forced to cease some aspect of our business operations, if, as a result of actual or threatened patent infringement claims, we are unable to enter into licenses on acceptable terms. Such a result would harm our business significantly.
There has been substantial litigation and other proceedings regarding patent and other intellectual property rights in the pharmaceutical and biotechnology industries. In addition to infringement claims against us, we may become a party to other patent litigation and other proceedings, including interference proceedings declared by the U.S. Patent and Trademark Office and opposition proceedings in the European Patent Office, regarding intellectual property rights with respect to our products and technology. Even if we prevail, the cost to us of any patent litigation or other proceeding could be substantial.
Some of our competitors may be able to sustain the costs of complex patent litigation more effectively than we can because they have substantially greater resources. In addition, any uncertainties resulting from any litigation could significantly limit our ability to continue our operations. Patent litigation and other proceedings may also absorb significant management time.
We may be subject to claims by third parties asserting that we or our employees have misappropriated their intellectual property, or claiming ownership of what we regard as our own intellectual property.
Many of our employees were previously employed at universities or other biotechnology or pharmaceutical companies, including our competitors or potential competitors. Although we try to ensure that our employees do not use the proprietary information or know-how of others in their work for us, we may be subject to claims that we or these employees have used or disclosed intellectual property, including trade secrets or other proprietary information, of any such employee's former employer. Litigation may be necessary to defend against these claims.
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In addition, while we typically require our employees and contractors who may be involved in the development of intellectual property to execute agreements assigning such intellectual property to us, we may be unsuccessful in executing such an agreement with each party who in fact develops intellectual property that we regard as our own. Our and their assignment agreements may not be self-executing or may be breached, and we may be forced to bring claims against third parties, or defend claims they may bring against us, to determine the ownership of what we regard as our intellectual property.
If we fail in prosecuting or defending any such claims, in addition to paying monetary damages, we may lose valuable intellectual property rights or personnel. Even if we are successful in prosecuting or defending against such claims, litigation could result in substantial costs, be a distraction to management, and divert time and resources away from other value-driving pursuits.
If we fail to comply with our obligations in our intellectual property licenses with third parties, we could lose license rights that are important to our business.
As part of our business, we have historically been a party to license agreements pursuant to which we license key intellectual property relating to certain products or product candidates. We expect to enter into additional licenses in the future. Such licenses impose various diligences, milestone payment, royalty, insurance and other obligations on us. If we fail to comply with these obligations, the licensor may have the right to terminate the license, in which event we might not be able to market any product or product candidate that is covered by the licensed patents.
We have not yet registered our trademarks in all of our potential markets, and failure to secure those registrations could adversely affect our business.
There is no guarantee that our trademark applications will be allowed for registration, or that our registered trademarks will be maintained or enforced. During trademark registration proceedings we may also receive rejections and, although we are given an opportunity to respond to these rejections, we may be unsuccessful in overcoming such rejections. In addition, in the U.S. Patent and Trademark Office and in comparable agencies in many foreign jurisdictions, third parties are given an opportunity to oppose pending trademark applications and to seek to cancel registered trademarks. Opposition or cancellation proceedings may be filed against our trademarks, and our trademarks may not survive such proceedings. If we do not secure registrations for our trademarks, we may encounter more difficulty in enforcing them against third parties than we otherwise would.
Risks Related to Employment, Sustainability and Governance Matters
Our future success depends on our ability to retain our Chief Executive Officer and other key personnel and to attract, retain and motivate qualified personnel.
We are highly dependent on Bradley L. Campbell, our President and Chief Executive Officer, and Simon Harford, our Chief Financial Officer, both of whom has significant pharmaceutical industry experience. The loss of the services of either of these individuals might impede the achievement of our research, development and commercialization objectives and materially adversely affect our business and we may not be able to replace them with candidates with similar background and experience in the event of the loss of their services. We do not maintain "key person" insurance on Mr. Campbell or on any of our other key personnel.
Recruiting and retaining qualified scientific, clinical, technical operations, and sales and marketing personnel will also be critical to our success. In addition, maintaining a qualified finance and legal department is key to our ability to meet our regulatory obligations as a public company and important in any potential business development or capital raising activities. Our industry has experienced a high rate of turnover in recent years. We may not be able to attract and retain these personnel on acceptable terms given the competition among numerous pharmaceutical and biotechnology companies for similar personnel, particularly in New Jersey, Philadelphia, and their surrounding areas. Although we believe we offer competitive salaries and benefits, we may have to increase spending in order to retain personnel. If we fail to retain our qualified personnel or replace them when they leave, we may be unable to recruit replacements without increased expense, if at all, or continue our development and commercialization activities.
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In addition, we rely on consultants and advisors, including scientific and clinical advisors, to assist us in formulating our research and development and commercialization strategy. Our consultants and advisors may be employed by employers other than us and may have commitments under consulting or advisory contracts with other entities that may limit their availability to us.
We expect to expand our development, regulatory and sales and marketing capabilities, and as a result, we may encounter difficulties in managing our growth, which could disrupt our operations.
As of December 31, 2025, we had 511 full-time employees. As our development and commercialization strategies evolve, we will need additional managerial, operational, sales, marketing, financial, technical operations and other resources. Our management, personnel and systems currently in place may not be adequate to support this future growth. We may not be able to effectively manage the expansion of our operations, which may result in weaknesses in our infrastructure, give rise to operational mistakes, loss of business opportunities, loss of employees, and reduced productivity among remaining employees. Future growth could require significant capital expenditures and may divert financial resources from other projects, such as the development of our existing or future product candidates, and we may not be able to replace key personnel in the event of turnover. Future growth would impose significant added responsibilities on members of management, including:
•managing the development and commercialization of any products or product candidates approved for marketing;
•managing our efforts to expand the patient population and indications of our products or of any product candidates for which we receive approval; successfully executing on our label expansion endeavors for any products or product candidates approved for marketing;
•overseeing our ongoing or future preclinical studies and clinical trials effectively, including the Galafold® clinical trials required under the accelerated approval regulations;
•identifying, recruiting, maintaining, motivating and integrating additional employees, including any sales and marketing personnel engaged in connection with the commercialization of any approved product;
•managing our internal development efforts effectively while complying with our contractual obligations to licensors, licensees, contractors and other third parties;
•managing any business development activities and successfully integrating any acquired assets or businesses;
•managing our collaboration partners and associated joint steering committees;
•managing any clinical or commercial collaborations with third parties;
•improving our managerial, development, operational and financial systems and procedures;
•developing our compliance infrastructure and processes to ensure compliance with regulations applicable to public companies;
•developing expertise in newly acquired or in-licensed technologies; and
•expanding our facilities.
As our operations expand, we will need to manage additional relationships with various strategic collaborators, suppliers and other third parties. Our future financial performance and our ability to commercialize our product candidates and to compete effectively will depend, in part, on our ability to manage any future growth effectively. To that end, we must be able to manage our development efforts and clinical trials effectively and hire, train and integrate additional management, administrative and sales and marketing personnel. We may not be able to accomplish these tasks, and our failure to accomplish any of them could prevent us from successfully growing our company.
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Our employees, independent contractors, principal investigators, CROs, consultants, agents, and vendors may engage in misconduct or other improper activities, including noncompliance with regulatory standards and requirements, which could cause significant liability for us and harm our reputation.
We are exposed to the risk that our employees, independent contractors, principal investigators, CROs, consultants, agents and vendors may engage in fraudulent conduct, harassment or other illegal activity. Misconduct by these parties could include intentional, reckless and/or negligent conduct or disclosure of unauthorized activities to us that violates:
•the FDA or similar regulations of foreign regulatory authorities, including those laws requiring the reporting of true, complete and accurate information to such authorities;
•manufacturing standards;
•federal and state healthcare fraud and abuse laws and regulations, anti-bribery and corruption laws, anti-discrimination and harassment laws, privacy and similar laws and regulations established and enforced by foreign regulatory authorities;
•laws that require the reporting of financial information or data accurately; or
•laws requiring the timely and accurate disclosure of material information to investors and analysts.
In particular, sales, marketing and business arrangements in the healthcare industry are subject to extensive laws and regulations intended to prevent fraud, kickbacks, self-dealing, bribery and corruption and other abusive practices. These laws and regulations may restrict or prohibit a wide range of pricing, discounting, marketing and promotion, sales commission, customer incentive programs and other business arrangements. Activities subject to these laws also involve the improper use of information obtained in the course of clinical trials, which could result in regulatory sanctions and serious harm to our reputation. We have adopted a Code of Business Conduct and Ethics, implemented a robust Enterprise Risk Management Program, have extensive Board of Directors oversight, and conduct comprehensive training, but it is not always possible to identify and deter misconduct by employees and other third parties, and the precautions we take to detect and prevent this activity may not be effective in controlling unknown or unmanaged risks or losses or in protecting us from governmental investigations or other actions or lawsuits stemming from a failure to be in compliance with such laws or regulations. If any such actions are instituted against us, and we are not successful in defending ourselves or asserting our rights, those actions could have a material adverse effect on our business and results of operations, including the imposition of civil, criminal and administrative penalties, damages, monetary fines, possible exclusion from participation in healthcare programs, contractual damages, reputational harm, diminished profits and future earnings, and curtailment of our operations, any of which could have a material adverse effect on our ability to operate our business and our results of operations.
If our enterprise risk program, global risk committee and other compliance methods are not effective, our business, financial condition and operating results may be adversely affected.
Our ability to identify, manage and respond to the various risks related to our business is largely dependent on our established and maintained compliance, risk, audit and reporting systems and procedures. The Board of Directors has ultimate responsibility for risk oversight of the company and carries out this duty through its various committees. Our Audit and Compliance Committee, Nominating and Corporate Governance Committee, Compensation and Leadership Development Committee and Science and Technology Committee have each been delegated oversight authority by the Board of Directors with respect to issues in their applicable areas of expertise. These committees are responsible for identifying, monitoring and reporting areas of concern to the full Board of Directors. At the company level, our senior management team similarly monitors risk through the Global Risk Committee. Membership of the Global Risk Committee consists primarily of key department heads who are asked to bring to such committee relevant items for discussion that they or their teams have identified at the numerous sub-committees these individuals chair or attend. The Global Risk Committee then uses this information to develop an Enterprise Risk Management Program, which identifies key risks, develops mitigation strategies for these risks, and reports material developments directly to the Audit and Compliance Committee on a quarterly basis, and the full Board of Directors on a yearly basis. Our international business segment also has its own companion committee which operates in substantially the same way as the Global Risk Committee, reporting key risks to the Global Risk Committee for inclusion in the Enterprise Risk Management Program.
If our policies, procedures, and compliance systems, including our Enterprise Risk Management Program and the Global Risk Committee are not effective, or if we are not successful in monitoring or evaluating the risks to which we are or may be
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exposed, our business, reputation, financial condition and operating results could be materially adversely affected. We cannot provide assurance that our policies and procedures will always be effective, or that our management, the Enterprise Risk Management Program or the Global Risk Committee would be able to identify any such ineffectiveness. If our compliance and risk management strategies are not effective, our business, financial condition and operating results may be adversely affected.
The increased focus on environmental emissions reporting by investors, governmental bodies and other stakeholders, as well as existing and proposed laws related to these topics, may adversely affect our business and reputation.
Companies are being increasingly judged by not just their financial performance, but, for those companies with substantial operations outside the U.S., also on business sustainability measures. These matters include, among others, (i) the company’s efforts, contributions to, or impacts on climate change, (ii) ethics and compliance with law, and (iii) the role of the company’s board of directors in supervising various sustainability issues. Additionally, in the healthcare, pharmaceutical and life sciences industries, the public’s ability to access our medicines is of particular importance.
Investment in funds that specialize in companies that perform well in these assessments are increasingly popular, and major institutional investors and advisors have publicly emphasized the importance of such measures to their investment decisions and recommendations. Investors who are focused on these matters may seek enhanced sustainability disclosures or to implement policies adverse to our business, and there can be no assurances that stockholders will not advocate, via proxy contests, media campaigns or other public or private means, for us to make corporate governance changes or engage in certain corporate actions.
Additionally, we are subject to an increasing number of laws and regulations promulgated by multiple countries and jurisdictions, including the U.S., E.U., and U.K., that require new and expansive climate-related disclosures, emissions tracking as it relates to both us and our supply chain partners, and, in certain jurisdictions, changes to our operations to ensure compliance with product packaging, energy consumption, and other country-specific environmental initiatives. In anticipation of these rules and regulations, we have convened a cross-functional, global climate task force, which reports to our Nominating and Corporate Governance Committee, and engaged external resources to develop and maintain new reporting and data collection processes and procedures. For many of these climate-related laws and regulations, compliance timelines are based on a combination of factors such as employee population size, yearly turnover, or total assets. As such, our estimated compliance dates are based on internal forecasts and projections that may change as our business continues to grow and evolve.
Compliance with any such new laws or regulations will be costly, time consuming, and, as a global commercial organization, require expenditure of our limited resources to be in compliance with the various standards across the jurisdictions in which we operate. Moreover, there can be no guarantee that we will be able to timely comply with such laws and regulations. Failure to adequately meet these new and upcoming disclosure requirements may affect the manner and locations in which we choose to conduct our business and could adversely affect our profitability and returns to our investors.
Our business may be impacted by actions of the U.S. government, including Executive Orders, policies, new legislation, and judicial decisions
Actions by the U.S. government may require us to change our business operations, resulting in unknown and unforeseeable impacts to our stakeholders, including patients, healthcare providers, and employees. Failure to comply with new policies, legislation, judicial decisions and executive orders could expose the Company to litigation or other government actions. There can be no assurance that our compliance with new government actions will provide sufficient mitigation.
Our business activities involve the use of hazardous materials, which require compliance with environmental and occupational safety laws regulating the use of such materials. If we violate these laws, we could be subject to significant fines, liabilities or other adverse consequences.
Our research and development programs involve the controlled use of hazardous materials, including microbial agents, corrosive, explosive and flammable chemicals and other hazardous compounds in addition to certain biological hazardous waste. Additionally, the activities of our third-party product manufacturers of our product, and of our product candidates if and when they reach commercialization, will also require the use of hazardous materials. Accordingly, we are subject to federal, state and local laws governing the use, handling and disposal of these materials. Although we believe that our safety procedures for handling and disposing of these materials comply in all material respects with the standards prescribed by local, state and federal regulations, we cannot completely eliminate the risk of accidental contamination or injury from these materials. In
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addition, although our collaborators have environmental compliance processes in place, and we include oversight of these processes in our business reviews, they may not ultimately comply with these laws. In the event of an accident or failure to comply with environmental laws, we could be held liable for damages that result, and any such liability could exceed our assets and resources or we could be subject to limitations or stoppages related to our use of these materials which may lead to an interruption of our business operations or those of our third-party contractors. While we believe that our existing insurance coverage is generally adequate for our normal handling of these hazardous materials, it may not be sufficient to cover pollution conditions or other extraordinary or unanticipated events. Furthermore, an accident could damage or force us to shut down our operations. Changes in environmental laws may impose costly compliance requirements on us or otherwise subject us to future liabilities and additional laws relating to the management, handling, generation, manufacture, transportation, storage, use and disposal of materials used in or generated by the manufacture of our products or related to our clinical trials. In addition, we cannot predict the effect that these potential requirements may have on us, our suppliers and contractors or our customers.
Our business could be adversely affected by the effects of health pandemics or epidemics, which could cause significant disruptions in our operations.
Health pandemics or epidemics have in the past and could again in the future result in quarantines, stay-at-home orders, remote work policies or other similar events that may disrupt businesses, delay our research and development programs and timelines, negatively impact productivity and increase risks associated with cybersecurity, the future magnitude of which will depend, in part, on the length and severity of the restrictions and other limitations. More specifically, these types of events may negatively impact personnel at third-party manufacturing facilities or the availability or cost of materials, which could disrupt our supply chain. In addition, impact on the operations of the FDA or other regulatory authorities could negatively affect our planned approval processes. Finally, economic conditions and business activity may be negatively impacted and may not recover as quickly as anticipated. The effects of epidemics and pandemic are highly uncertain and subject to change. If we are not able to respond to and manage the impact of such events effectively, our business, operating results, financial condition and cash flows could be adversely affected.
General Risk Factors
Our business and operations would suffer in the event of computer system failures or security breaches.
Despite the implementation of security measures, our internal computer systems, and those of our CROs, contract manufacturing organizations and other third parties on which we rely, are vulnerable to damage from computer viruses, unauthorized access, ransomware attacks and other security breaches, natural disasters, terrorism, war and telecommunication and electrical failures. System failures, accidents or security breaches could cause interruptions in our operations and could result in a material disruption of our clinical activities and business operations, in addition to possibly requiring substantial expenditures of resources to remedy. If such an event were to occur and cause interruptions to our operations, it could result in a material disruption to the commercialization of our products and our product candidate development programs. For example, the loss of clinical trial data from completed or ongoing clinical trials could result in delays in our regulatory approval efforts and significantly increase our costs to recover or reproduce the data. To the extent that any disruptions or security breach were to result in a loss or damage to our data or applications, or inappropriate disclosure of confidential or proprietary information, we could incur significant unexpected losses, expenses and liabilities, we could face litigation or suffer reputational harm and the further development of our product candidates could be delayed.
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In addition, cybersecurity threats and reported incidents are increasing in their frequency, sophistication and intensity, including as a result of ongoing military conflicts, certain U.S. foreign relations, and increased remote work arrangements, and are becoming increasingly difficult to detect, particularly when they impact vendors, customers or suppliers, and other companies in our supply chain. Cybersecurity threats or incidents may include the deployment of malware via emails disguised to look legitimate or the use of social engineering to obtain employee access credentials to the company’s computer network and systems, as well as various other schemes and approaches designed to breach company cyber defenses. Once access has been obtained, the illegitimate actor can steal sensitive information, install ransomware requiring a large financial outlay to recover company systems and files, or wreak havoc in a variety of different and creative ways. While we have robust detection, mitigation, response and recovery protocols in place, there is no guarantee that these will be effective in preventing disruptions to our operations and adequately safeguard confidential, proprietary or sensitive information from misappropriation or corruption. Our key business partners, manufacturers and vendors face these same risks and a successful attack on their systems could have a similar negative impact to our business and operations. Moreover, SEC reporting requirements mandate specific disclosures in the event of a material cybersecurity incident. As such, we may have to report certain incidents that could result in reputational harm and loss of investor, customer and patient confidence even if our cybersecurity defenses are ultimately effective in staving off such incidents. To date and to our knowledge, we have not experienced a material cybersecurity incident.
We may use artificial intelligence in our business, and challenges with properly managing its use could adversely affect our business.
We may incorporate artificial intelligence (“AI”) solutions into our business, and applications of AI may become important in our operations over time. Our competitors or other third parties may incorporate AI into their businesses more quickly or more successfully than us, which could impair our ability to compete effectively and adversely affect our results of operations.
There are also significant risks involved in developing and deploying AI, and there can be no assurance that the usage of AI will enhance our products or the development of our product candidates or be beneficial to our business, including our efficiency or profitability. For example, our AI-related efforts, particularly those related to generative AI, could subject us to risks related to harmful content, inaccuracies, bias, discrimination, toxicity, intellectual property infringement or misappropriation, defamation, data privacy, cybersecurity, and sanctions and export controls, among others. It is also uncertain how various laws will apply to content generated by AI. We are subject to the risks of new or enhanced governmental or regulatory scrutiny, litigation, or other legal liability, ethical concerns, negative consumer perceptions as to automation and AI, or other complications that could adversely affect our business, reputation, or financial results.
AI’s rapid development is the subject of evolving review by various U.S. governmental and regulatory agencies, and other foreign jurisdictions are applying, or are considering applying, their intellectual property, cybersecurity, data protection and other laws to AI, and/or are considering general legal frameworks on AI. We may not be able to timely comply with these frameworks and, if such regulatory actions are contrary to our use of AI, they may require us to expend our limited resources to adjust our use of AI accordingly.
We may acquire or divest assets or businesses, or form collaborations or make investments in other companies or technologies that could harm our operating results, dilute our stockholders' ownership, increase our debt, or cause us to incur significant expense.
As part of our business strategy, we may continue to pursue acquisitions or licenses of assets or businesses, or strategic alliances and collaborations, to expand our existing technologies and operations. We may not identify or complete these transactions in a timely manner, on a cost-effective basis, or at all despite a substantial outlay of resources in pursuing such transactions, and we may not realize the anticipated benefits of any such transaction, any of which could have a detrimental effect on our financial condition, results of operations, and cash flows. We may not be able to find suitable acquisition or licensing candidates, and if we make any acquisitions, we may not be able to, the extent applicable, successfully complete any on-going or future clinical trials to obtain the data necessary for regulatory approval or, if already approved, successfully commercialize such acquisition to the extent forecasted or otherwise expected. Additionally, integration of such acquisitions into our existing business may lead to the incurrence of additional debt, issuance of equity, or assumption of unknown or contingent liabilities in connection therewith. Integration of an acquired company or assets may also disrupt ongoing operations, require the hiring of additional personnel and the implementation of additional internal systems and infrastructure, especially the acquisition of commercial assets, and require management resources that would otherwise focus on developing
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our existing business. We may not be able to find suitable collaboration partners or identify other investment opportunities, and we may experience losses related to any such investments.
To finance any acquisitions, licenses or collaborations, we may choose to issue debt or shares of our common stock as consideration. Any such issuance of shares would dilute the ownership of our stockholders. If the price of our common stock is low or volatile, we may not be able to acquire other assets or companies or fund a transaction using our stock as consideration. Alternatively, it may be necessary for us to raise additional funds for acquisitions through public or private financings. Additional funds may not be available on terms that are favorable to us, or at all.
In addition, we may divest or license all or a portion of certain businesses and/or facilities, joint venture or minority equity investment interests, subsidiaries, distributorships, or product categories, which could cause a decline in revenue or profitability and may make our financial results more volatile. We may be unable to complete any such divestiture or license on terms favorable to us, within the expected timeframes, or at all despite a substantial outlay of resources in pursuing such divesture or license. We may have continued financial exposure to divested or licensed businesses following the completion of any such transaction, including increased costs due to potential litigation, contingent liabilities and indemnification of the buyer or licensee related to, among other things, lawsuits, regulatory matters or tax liabilities. Such divestitures or licenses may also divert management’s attention from our core businesses and lead to potential issues with employees, customers or suppliers.
Provisions in our corporate charter documents and under Delaware law could make an acquisition of us, which may be beneficial to our stockholders, more difficult and may prevent attempts by our stockholders to replace or remove our current management.
Provisions in our corporate charter and our bylaws may discourage, delay or prevent a merger, acquisition or other change in control of us that stockholders may consider favorable, including transactions in which our stockholders might otherwise receive a premium for their shares. These provisions could also limit the price that investors might be willing to pay in the future for shares of our common stock, thereby depressing the market price of our common stock. In addition, these provisions may frustrate or prevent any attempts by our stockholders to replace or remove our current management by making it more difficult for stockholders to replace members of our board of directors. Because our board of directors is responsible for appointing the members of our management team, these provisions could in turn affect any attempt by our stockholders to replace current members of our management team. Among others, these provisions:
•establish a classified board of directors, and, as a result, not all directors are elected at one time;
•allow the authorized number of our directors to be changed only by resolution of our board of directors;
•limit the manner in which stockholders can remove directors from our board of directors;
•establish advance notice requirements for stockholder proposals that can be acted on at stockholder meetings and nominations to our board of directors;
•require that stockholder actions must be effected at a duly called stockholder meeting and prohibit actions by our stockholders by written consent;
•limit who may call stockholder meetings;
•authorize our board of directors to issue preferred stock, without stockholder approval, which could be used to institute a "poison pill" that would work to dilute the stock ownership of a potential hostile acquirer, effectively preventing acquisitions that have not been approved by our board of directors; and
•require the approval of the holders of at least 67% of the outstanding voting stock to amend or repeal certain provisions of our charter or bylaws.
Moreover, because we are incorporated in Delaware, we are governed by the provisions of Section 203 of the Delaware General Corporation Law, which prohibits a person who owns in excess of 15% of our outstanding voting stock from merging or combining with us for a period of three years after the date of the transaction in which the person acquired in excess of 15% of our outstanding voting stock, unless the merger or combination is approved in a prescribed manner.
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Unfavorable global economic conditions, whether brought about by material global crises, health epidemics, military conflicts or war, geopolitical and trade disputes or other factors, may adversely affect our business and financial results.
Our business is sensitive to global economic conditions, which can be adversely affected by public health crises and epidemics, political and military conflict, trade and other international disputes, significant natural disasters (including as a result of climate change) or other events that disrupt macroeconomic conditions.
For example, climate change could present risks to our operations, including an adverse impact on global temperatures, weather patterns and the frequency and severity of extreme weather and natural disasters. Natural disasters and extreme weather conditions, such as a hurricane, tornado, earthquake, wildfire or flooding, may pose physical risks to our facilities, employees, customers, patients and disrupt the operation of our supply chain and increase operational costs.
Additionally, trade policies and geopolitical disputes (including as a result of PRC-Taiwan relations) and other international conflicts can result in tariffs, sanctions and other measures that restrict international trade, and can materially adversely affect our business, particularly if these measures occur in regions where we source our components or raw materials. Tensions between the U.S. and PRC have led to a series of tariffs being imposed by the U.S. on imports from PRC mainland, as well as other business restrictions. Tariffs increase the costs of the components and raw materials we source. Countries may also adopt other measures, such as controls on imports or exports of goods, technology or data, that could adversely impact our operations and supply chain. As these tensions continue to rise, more targeted approaches by the U.S. or Chinese governments on certain products, industries or companies (including WuXi, the primary supplier of one of our products) could significantly impact our ability to effectively manufacture and distribute our products, including Pombiliti® + Opfolda®, materially impacting our ability to meet patient demands or financial forecasts. With the new presidential administration in the U.S., additional and higher tariffs may be imposed on goods imported from PRC and other countries which could increase the cost of goods needed to commercialize our products and continue development of our product candidates. Further, such actions by the U.S. could result in retaliatory action by those countries which could impact our ability to profitably commercialize our products in those jurisdictions. As a result, our business, operations, and financial condition could be materially harmed.
Further, recent global events have adversely affected and are continuing to adversely affect workforces, organizations, economies, and financial markets globally, leading to economic downturns, inflation, and increased market volatility. Military conflicts and wars (such as the ongoing conflicts between Russia and Ukraine, Israel and Hamas, and the Red Sea crisis and its impact on shipping and logistics), terrorist attacks, instability in Venezuela, other geopolitical events, high inflation, increasing interest rates, bank failures and associated financial instability and crises, and supply chain issues can cause exacerbated volatility and disruptions to various aspects of the global economy. The uncertain nature, magnitude, and duration of hostilities stemming from such conflicts, including the potential effects of sanctions and counter-sanctions, or retaliatory cyber-attacks on the world economy and markets, have contributed to increased market volatility and uncertainty, which could have an adverse impact on macroeconomic factors that affect our business and operations.
Significant changes or developments in U.S. laws or policies, including changes in U.S. trade policies and tariffs and the reaction of other countries thereto, may have a material adverse effect on our business and financial statements.
As a biotechnology company dedicated to discovering, developing, and delivering novel medicines for patients with rare diseases, we operate in a highly regulated space, sourcing products and delivering our medicines around the world. As such, we are sensitive to governmental changes that affect the regulatory pathway, and approval status, of our medicines, as well as our ability to effectively, and in a cost-efficient manner, produce and distribute these medicines. Our pricing and reimbursement strategy considers, among other things, the cost of bringing a particular treatment to market and the cost of goods incurred to produce the final product. Accordingly, significant changes or developments in U.S. laws and policies, such as laws and policies surrounding international trade, foreign affairs, manufacturing and development and investment in the territories and countries where we or our customers operate, can materially adversely affect our business and financial statements. The current presidential administration has indicated a number of potential reforms that could have a material adverse effect on our business, future revenue, and results of operations, including changes impacting the FDA and the scope of its authority, imposition of significant tariffs on numerous countries (including the PRC, which is a primary supplier of Pombiliti®), instituting a "most favored nation" policy requiring pharmaceutical companies to lower U.S. drug prices to match the lowest prices such drugs are sold for in other developed countries, and other, unforeseen developments that typically accompany a new presidential administration While it is impossible to predict future legislative or administrative actions, similar trade restrictions and regulatory changes could result in additional requirements for commercialization for our approved products and
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development of our product candidates, an increase in the cost of goods required to make our medicines, or otherwise impair, impede or frustrate our ability to deliver medicines globally.
The price of our common stock may be volatile and fluctuate substantially, which could result in substantial losses for purchasers of our common stock.
The market price of our common stock is highly volatile and may be subject to wide fluctuations in response to numerous factors, some of which are beyond our control. In addition to the factors discussed in this Annual Report on Form 10-K, these factors include:
•the perceived likelihood of the completion of the pending transaction with BioMarin;
•the success of competitive products or technologies;
•regulatory actions with respect to our products or product candidates or our competitors' products or product candidates;
•actual or anticipated changes in our growth rate relative to our competitors;
•the outcome of any patent infringement or other litigation that may be brought against us or we may bring against others;
•announcements by us or our competitors of significant acquisitions, mergers, strategic collaborations, joint ventures, collaborations or capital commitments;
•results of clinical trials of our product candidates or those of our competitors;
•regulatory or legal developments in the E.U., U.K., U.S. and other countries;
•developments or disputes concerning patent applications, issued patents or other proprietary rights;
•the recruitment or departure of key personnel;
•the level of expenses related to our product or any of our product candidates or clinical development programs;
•actual or anticipated variations in our quarterly operating results;
•the number and characteristics of our efforts to in-license or acquire additional product candidates or products;
•introduction of new products or services by us or our competitors;
•failure to meet the estimates and projections of the investment community or that we may otherwise provide to the public;
•actual or anticipated changes in estimates as to financial results, development timelines or recommendations by securities analysts;
•variations in our financial results or those of companies that are perceived to be similar to us;
•fluctuations in the valuation of companies perceived by investors to be comparable to us;
•share price and volume fluctuations attributable to inconsistent trading volume levels of our shares;
•announcement or expectation of additional financing efforts;
•sales of our common stock by us, our insiders or our other stockholders;
•changes in accounting practices;
•lawsuits and other claims asserted against us;
•changes in the structure of healthcare payment systems;
•market conditions in the pharmaceutical and biotechnology sectors;
•general economic, industry and market conditions;
•publication of research reports about us, our competitors or our industry, or positive or negative recommendations or withdrawal of research coverage by securities or industry analysts;
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•other events or factors, many of which are beyond our control; and
•the other factors described in this "Risk Factors" section.
In addition, the stock market in general, and pharmaceutical and biotechnology companies in particular, have experienced extreme price and volume fluctuations that have often been unrelated or disproportionate to the operating performance of these companies. Broad market and industry factors may negatively affect the market price of our common stock, regardless of our actual operating performance. The realization of any of the above risks or any of a broad range of other risks stated above could have a material adverse effect on the market price of our common stock.
As we operate in the pharmaceutical and biotechnology industry, we are especially vulnerable to these factors to the extent that they affect our industry or our products. In the past, securities class action litigation has often been initiated against companies following periods of volatility in their stock price. This type of litigation could result in substantial costs and divert our management's attention and resources, and could also require us to make substantial payments to satisfy judgments or to settle litigation.
A significant portion of our total outstanding shares may be sold into the market. This could cause the market price of our common stock to drop significantly, even if our business is doing well.
Sales of a substantial number of shares of our common stock in the public market could occur at any time. These sales, or the perception in the market that the holders of a large number of shares intend to sell shares, could reduce the market price of our common stock. Certain holders of our common stock have rights, subject to some conditions, to require us to file registration statements covering their shares or to include their shares in registration statements that we may file for ourselves or other stockholders. We also have registered on Form S-8 registration statements all shares of common stock that we may issue under our equity compensation plans. As a result, these shares can be freely sold in the public market upon issuance, subject to volume limitations applicable to affiliates. In addition, certain of our employees, executive officers and directors have entered into, or may enter into, Rule 10b5-1 plans providing for sales of shares of our common stock from time to time. Under a Rule 10b5-1 plan, a broker executes trades pursuant to parameters established by the employee, director or officer when entering into the plan, without further direction from the employee, officer or director. A Rule 10b5-1 plan may be amended or terminated in some circumstances. Our employees, executive officers and directors may also buy or sell additional shares outside of a Rule 10b5-1 plan when they are not in possession of material, nonpublic information. In November 2022, we announced an "at-the-market offering" under which we may offer and sell shares of our common stock having an aggregate offering amount of up to $250,000,000. Additionally, in October 2023, we entered into a securities purchase agreement with Blackstone for the private placement of 2,467,104 shares of our common stock, at a purchase price of $12.16 per share.
We may fail to qualify for continued listing on The NASDAQ Global Market which could make it more difficult for investors to sell their shares.
Our common stock is listed on The NASDAQ Global Market ("NASDAQ"). As a NASDAQ listed company, we are required to satisfy the continued listing requirements of NASDAQ for inclusion in the Global Market to maintain such listing, including, among other things, the maintenance of a minimum closing bid price of $1.00 per share and stockholders' equity of at least $10.0 million. There can be no assurance that we will be able to maintain compliance with the continued listing requirements or that our common stock will not be delisted from NASDAQ in the future. If our common stock is delisted by NASDAQ, we could face significant material adverse consequences, including:
•a limited availability of market quotations for our securities;
•reduced liquidity with respect to our securities;
•a determination that our shares are a "penny stock," which will require brokers trading in our shares to adhere to more stringent rules, possibly resulting in a reduced level of trading activity in the secondary trading market for our shares;
•a limited amount of news and analyst coverage for our company; and
•a decreased ability to issue additional securities or obtain additional financing in the future.
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If securities or industry analysts do not publish research or reports or publish unfavorable research about our business, the price of our common stock and trading volume could decline.
The trading market for our common stock depends in part on the research and reports that securities or industry analysts publish about us or our business. If securities or industry analysts do not initiate or continue coverage of us, the trading price for our common stock would be negatively affected. In the event we obtain securities or industry analyst coverage, if one or more of the analysts who covers us downgrades our common stock, the price of our common stock would likely decline. If one or more of these analysts ceases to cover us or fails to publish regular reports on us, interest in the purchase of our common stock could decrease, which could cause the price of our common stock or trading volume to decline.
We have broad discretion in the use of our cash and cash equivalents and may not use them effectively.
We have broad discretion in the use of our cash and cash equivalents, and investors must rely on the judgment of our management regarding the use of our cash and cash equivalents. Our management may not use cash and cash equivalents in ways that ultimately increase the value of your investment. Our failure to use our cash and cash equivalents effectively could result in financial losses that could have a material adverse effect on our business, cause the price of our common stock to decline and delay the development of our products and product candidates. Pending their use, we may invest our cash and cash equivalents in short-term or long-term, investment-grade, interest-bearing securities. These investments may not yield favorable returns. If we do not invest or apply our cash and cash equivalents in ways that enhance stockholder value, we may fail to achieve expected financial results, which could cause the price of our common stock to decline.
If we are unable to protect the confidentiality of our trade secrets, our business and competitive position would be harmed.
In addition to seeking patents for some of our technology and products, we also rely on trade secrets, including unpatented know-how, technology and other proprietary information, to maintain our competitive position. We seek to protect these trade secrets, in part, by entering into non-disclosure and confidentiality agreements with parties who have access to them, such as our employees, corporate collaborators, outside scientific collaborators, contract manufacturers, consultants, advisors and other third parties. We also enter into confidentiality and invention or patent assignment agreements with our employees and consultants. However, we cannot guarantee that we have executed these agreements with each party that may have or have had access to our trade secrets or that the agreements we have executed will provide adequate protection. Any party with whom we have executed such an agreement may breach that agreement and disclose our proprietary information, including our trade secrets, and we may not be able to obtain adequate remedies for such breaches. Enforcing a claim that a party illegally disclosed or misappropriated a trade secret is difficult, expensive and time-consuming, and the outcome is unpredictable. In addition, some courts inside and outside the U.S. are less willing or unwilling to protect trade secrets. If any of our trade secrets were to be lawfully obtained or independently developed by a competitor, we would have no right to prevent them, or those to whom they communicate it, from using that technology or information to compete with us. If any of our trade secrets were to be obtained or independently developed by a competitor, our competitive position would be harmed.
Litigation may adversely affect our business, financial condition, results of operations or liquidity.
Our business is subject to the risk of litigation by employees, consumers, vendors, competitors, intellectual property rights holders, stockholders, government agencies and others through private actions, class actions, administrative proceedings, regulatory actions, Hatch-Waxman or other litigation. For example, we and certain of our current and former officers have previously been parties to securities class action lawsuits against us, all of which have been settled or dismissed, and we were recently involved in Hatch-Waxman litigation that concluded in December 2025. The outcome of litigation, particularly class action lawsuits, regulatory actions and intellectual property claims, is difficult to assess or quantify. Plaintiffs in these types of lawsuits may seek recovery of very large or indeterminate amounts, and the magnitude of the potential loss relating to these lawsuits may remain unknown for substantial periods of time. In addition, certain of these lawsuits, if decided against us or settled by us, may result in liability material to our Consolidated Financial Statements as a whole or may negatively affect our operating results if changes to our business operation are required. The cost to prosecute or defend litigation may be significant. There also may be adverse publicity associated with litigation that could negatively affect customer perception of our business, regardless of whether the allegations are valid or whether we are ultimately found liable. As a result, litigation may adversely affect our business, financial condition, results of operations or liquidity.
We may be exposed to employment-related claims and losses which could have an adverse effect on our business.
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As we continue to increase the size of our workforce, the risk of potential employment-related claims will also increase. As such, we may be subject to claims, allegations or legal proceedings related to employment matters including, but not limited to, discrimination, harassment (sexual or otherwise), wrongful termination or retaliation, local, state or federal labor law violations, injury, and wage violations. In the event we are subject to one or more employment-related claims, allegations or legal proceedings, we may incur substantial costs, losses or other liabilities in the defense, investigation, settlement or other disposition of such claims. In addition to the economic impact, we may also suffer reputational harm as a result of such claims, allegations and legal proceedings and the investigation, defense and prosecution of such claims, allegations and legal proceedings could cause substantial disruption in our business and operations. While we do have policies and procedures in place to reduce our exposure to these risks, there can be no assurance that such policies and procedures will be effective or that we will not be exposed to such claims, allegations or legal proceedings.
Item 1B. UNRESOLVED STAFF COMMENTS
None.
Item 1C. CYBERSECURITY
The Company’s Board of Directors (the “Board”) recognizes the critical importance of maintaining the trust and confidence of our customers, clients, business partners and employees. The Board is actively involved in oversight of the Company’s Enterprise Risk Management Program (“ERMP”), and cybersecurity represents an important component of the Company’s overall approach to enterprise risk management. The Company’s cybersecurity policies, standards, processes and practices are integrated into the Company’s ERMP and are based on recognized frameworks established by the National Institute of Standards and Technology and other applicable industry standards. In general, the Company seeks to address cybersecurity risks through a systematic, cross-functional approach that is focused on preserving the confidentiality, security and availability of the information that the Company collects and stores by identifying, preventing and mitigating cybersecurity threats and responding to cybersecurity incidents if they should occur.
Risk Management and Strategy
As one of the critical elements of the Company’s overall enterprise risk management approach, the Company’s cybersecurity program is focused on the following key areas:
•Governance: As discussed in more detail under the heading “Governance,” the Board’s oversight of cybersecurity is delegated to the Audit and Compliance Committee of the Board, which oversees the Company’s entire ERMP, reporting up to the full board on a periodic basis. The Company’s Chief Information Officer (“CIO”), the Chief Compliance Officer and other members of management regularly report to the Audit and Compliance Committee, with cybersecurity representing a standing meeting agenda topic.
•Collaborative Approach: The Company has implemented a systematic, cross-functional approach to identifying, preventing and mitigating cybersecurity threats and incidents, while also implementing controls and procedures that provide for the prompt escalation of certain cybersecurity incidents so that decisions regarding the public disclosure and reporting of such incidents can be made by management in a timely manner.
Technical Safeguards: The Company deploys technical safeguards that are designed to protect the Company’s information systems from cybersecurity threats, including firewalls, intrusion prevention and detection systems, anti-malware functionality and access controls, 24x7 security monitoring, and other controls which are evaluated and improved through vulnerability assessments and cybersecurity threat intelligence.
•Incident Response and Recovery Planning: The Company has established and maintains systematic incident response and recovery plans that address the Company’s response to a cybersecurity incident, and such plans are tested and evaluated on a periodic basis.
•Third-Party Risk Management: The Company maintains a systematic, risk-based approach to identifying and overseeing cybersecurity risks presented by third parties, including vendors, service providers and other external users of the Company’s systems, as well as the systems of third parties that could adversely impact our business in the event of a cybersecurity incident affecting those third-party systems.
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•Education and Awareness: The Company provides regular, mandatory cybersecurity training for all personnel as a means to equip the Company’s workforce with effective tools to recognize, address and communicate potential or actual threats to the Company’s cybersecurity systems. Moreover, these trainings also allow Company personnel to remain up-to-date with evolving information security policies, standards, processes and best practices.
The Company engages in the periodic assessment and testing of the Company’s policies, standards, processes and practices that are designed to address cybersecurity threats and incidents. These efforts include a wide range of activities, including audits, assessments, tabletop exercises, threat modeling, vulnerability testing and other exercises focused on evaluating the effectiveness of our cybersecurity measures and planning. The Company has also engaged third parties to perform assessments on our cybersecurity measures, including information security maturity assessments, audits and independent reviews of our information security control environment and operating effectiveness. The results and findings of these exercises are reported to the Audit and Compliance Committee, who in turn updates the Board as appropriate. Management will then evaluate such findings and, with input from the Audit and Compliance Committee, take the appropriate steps to adjust the Company’s cybersecurity policies, standards, processes and practices, as may be applicable, to strengthen or address any weaknesses, gaps or findings as the case may be.
Governance
The Board has delegated their oversight of cybersecurity to the Company’s Audit and Compliance Committee which oversees the entire ERMP process. As detailed above, cybersecurity is a standing agenda topic for the Audit and Compliance Committee which receives regular presentations and reports from the Company’s CIO on cybersecurity risks, detection protocols, disaster recovery readiness, the threat environment, recent developments in the cybersecurity space (including known incidents affecting the Company or key Company suppliers), evolving standards, vulnerability assessments, third-party and independent reviews, technological trends and information security considerations arising with respect to the Company’s peers and third parties. Under the current cybersecurity framework, the Audit and Compliance Committee receives prompt and timely information regarding any cybersecurity incident that meets established reporting thresholds, as well as ongoing updates regarding any such incident. board informed, as may be appropriate, until any such threat has been addressed to their satisfaction. Additionally, cybersecurity is also a standing agenda topic for the Global Risk Committee, with periodic updates to the Executive Committee.
The CIO, in coordination with the Chief Executive Officer, Chief Financial Officer, Chief Compliance Officer, Chief Legal Officer, and Chief People Officer works collaboratively across the Company to implement a program designed to protect the Company’s information systems from cybersecurity threats and to promptly respond to any cybersecurity incidents in accordance with the Company’s incident response and recovery plans. To facilitate the success of the Company’s cybersecurity risk management program, multidisciplinary teams, comprised of our cybersecurity experts, are deployed throughout the Company to identify and address cybersecurity threats and to respond to cybersecurity incidents. The CIO has served in various leadership roles in information technology and information security for over 25 years, including serving as the vice president of information technology, with direct responsibility over the cyber security program, for a large publicly-traded company and as the chief information security officer of several large healthcare organizations. The CIO holds a Certified Information Systems Security Professional certification, an undergraduate degree in engineering, an MBA and a PhD in engineering.
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Item 2. PROPERTIES
The following table contains information about our current significant leased properties as of December 31, 2025.
| Location | Approximate Square Feet | Use | Lease expiry date (1) | |||||||||||||||||
Philadelphia, Pennsylvania, U.S. | 50,816 | Office and laboratory | September 2034 | |||||||||||||||||
| Marlow, United Kingdom | 36,796 | Office | August 2028 | |||||||||||||||||
| Princeton, New Jersey, U.S. | 29,972 | Office | January 2034 | |||||||||||||||||
______________________________
(1) Includes renewal options on leases which we are reasonably certain to exercise.
In addition to the above, we also maintain offices in other U.S. and international jurisdictions in which we operate. We believe that our current office and laboratory facilities are adequate and suitable for our current and anticipated needs. We believe that, to the extent required, we will be able to lease or buy additional facilities at commercially reasonable rates.
Item 3. LEGAL PROCEEDINGS
The information called for by this item is incorporated herein by reference to the information set forth in " -— Note 16. Legal Proceedings” in the Notes to Consolidated Financial Statements included in Item 8 of this Report.
Item 4. MINE SAFETY DISCLOSURES
None.
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PART II
Item 5. MARKET FOR THE REGISTRANT'S COMMON EQUITY, RELATED STOCKHOLDER MATTERS, AND ISSUER PURCHASES OF EQUITY SECURITIES
Market for Our Common Stock
Our common stock has been traded on the NASDAQ Global Market under the symbol "FOLD" since May 31, 2007. Prior to that time, there was no public market for our common stock. The closing price for our common stock as reported by the NASDAQ Global Market on February 12, 2026 was $14.33 per share. As of February 12, 2026, there were 15 holders of record of our common stock.
Dividends
We have never declared or paid any dividends on our capital stock. We currently intend to retain any future earnings to finance the development and growth of our business. We do not intend to declare or pay cash dividends to our stockholders in the foreseeable future.
Recent Sales of Unregistered Securities
None.
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Performance Graph
The following performance graph compares the cumulative total return on our common stock during the last five fiscal years with the NASDAQ Composite Index (U.S.) and the NASDAQ Biotechnology Index during the same period. The graph shows the value at the end of each of the last five fiscal years of $100 invested in our common stock. Pursuant to applicable SEC rules, all values assume reinvestment of the full amount of all dividends, however no dividends have been declared on our common stock to date. The stockholder return shown on the graph below is not necessarily indicative of future performance, and we do not make or endorse any predictions as to future stockholder returns.
| 12/31/2020 | 12/31/2021 | 12/31/2022 | 12/31/2023 | 12/31/2024 | 12/31/2025 | ||||||||||||||||||||||||||||||
| Amicus Therapeutics, Inc. | $100 | $50 | $52 | $62 | $41 | $62 | |||||||||||||||||||||||||||||
| NASDAQ Composite | $100 | $121 | $81 | $115 | $150 | $180 | |||||||||||||||||||||||||||||
| NASDAQ Biotechnology | $100 | $99 | $88 | $94 | $91 | $120 | |||||||||||||||||||||||||||||
The stock price performance included in this graph is not necessarily indicative of future stock price performance.
Issuer Purchases of Equity Securities
The following table provides certain information with respect to purchase of our common stock during the three months ended December 31, 2025:
| Period | Total Number of Shares Purchased (1) | Average Price Paid per Share | Total Number of Shares Purchased as Part of Publicly Announced Plans or Programs | Maximum Number (or Approximate Dollar Value) of Shares That May Yet Be Purchased Under the Plans or Programs | ||||||||||||||||||||||
| October 1, 2025 through October 31, 2025 | 20,767 | $ | 8.35 | — | — | |||||||||||||||||||||
| November 1, 2025 through November 30, 2025 | 23,325 | $ | 9.28 | — | — | |||||||||||||||||||||
| December 1, 2025 through December 31, 2025 | 298,936 | $ | 13.82 | — | — | |||||||||||||||||||||
| Total | 343,028 | $ | 13.18 | — | — | |||||||||||||||||||||
______________________________
(1) Represents shares of common stock withheld to satisfy taxes associated with the vesting of restricted stock awards.
Item 6. [RESERVED]
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Item 7. MANAGEMENT'S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS
Overview
We are a leading, global biotechnology company with a clear and compelling mission to develop and deliver transformative medicines for people living with rare diseases. With extraordinary patient focus, we strive to redefine expectations in rare disease. Our two marketed therapies are Galafold®, the first oral monotherapy for people living with Fabry disease who have amenable genetic variants, and Pombiliti® + Opfolda®, a novel two-component treatment designed to improve uptake of active enzyme into key disease relevant tissues for adults living with late-onset Pompe disease.
Galafold® (also referred to as "migalastat") is approved in over 40 countries around the world, including the United States ("U.S."), European Union ("E.U."), United Kingdom ("U.K."), and Japan. Additionally, Galafold® has been granted orphan drug designation in the U.S., E.U., U.K., Japan, and several other countries.
Pombiliti® + Opfolda® (also referred to as "cipaglucosidase alfa-atga/miglustat") is approved in the U.S., the E.U., the U.K., Canada, Australia, Switzerland, and Japan. Multiple regulatory submissions and reimbursement processes with global health authorities are currently underway. Additionally, Pombiliti® + Opfolda® has been granted orphan drug designation or status in the U.S., U.K., Switzerland and Japan and data exclusivity in the E.U.
On April 30, 2025, we entered into an exclusive license agreement with Dimerix Bioscience Pty Limited ("Dimerix") for the United States commercialization rights of Dimerix' Phase 3 drug candidate, DMX-200 for treatment of Focal Segmental Glomerulosclerosis ("FSGS") and other indications. In exchange for these rights, we paid Dimerix an upfront payment of $30 million. We will be obligated to pay Dimerix for certain success-based development and regulatory milestones for FSGS of up to a maximum aggregate amount of $75 million, regulatory milestones for other indications of up to a maximum aggregate amount of $40 million, commercial milestones of up to a maximum aggregate amount of $445 million, and tiered royalties of DMX-200 net sales in the U.S. ranging from the low-teens to low-twenties. Dimerix will continue to fund and execute the ACTION3 study, and we will be responsible for submission and maintenance of the regulatory dossier in the United States, as well as all costs of commercialization activities. Additionally, we will have the exclusive rights to develop DMX-200 in other future indications in the United States. Amicus and Dimerix have formed a Joint Steering Committee to align the development and commercialization of DMX-200 in FSGS in U.S.
On December 19, 2025, we entered into a Merger Agreement with BioMarin and its wholly owned acquisition subsidiary. Under the terms of the Merger Agreement, and in accordance with the DGCL, if the Merger is completed, BioMarin's wholly owned acquisition subsidiary will merge with and into Amicus, and Amicus will continue as the surviving corporation as a wholly owned subsidiary of BioMarin. In addition, all shares of our common stock outstanding immediately prior to closing (other than Excluded Shares or Dissenting Shares (as defined in the Merger Agreement)) will be cancelled and converted into the right to receive $14.50 per share in cash, without interest and subject to any applicable withholding of taxes. Following completion of the Merger, shares of our common stock will no longer be publicly listed. Refer to "— Note 15. Merger Agreement," in our Notes to Consolidated Financial Statements for additional information.
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Consolidated Results of Operations
The following discussion should be read in conjunction with the Consolidated Financial Statements and related notes included elsewhere in this report. The following section generally discusses 2025 and 2024 items and year-to-year comparisons between 2025 and 2024. Discussions of 2023 items and year-to-year comparisons between 2024 and 2023 that are not included in this Form 10-K can be found in Part II, Item 7 of the Company’s Annual Report on Form 10-K for the fiscal year ended December 31, 2024, which comparisons are hereby incorporated by reference.
Year Ended December 31, 2025 Compared to Year Ended December 31, 2024
The following table provides selected financial information of our operations:
| Years ended December 31, | ||||||||||||||||||||
| (in thousands) | 2025 | 2024 | Change | |||||||||||||||||
| Net product sales | $ | 634,210 | $ | 528,295 | $ | 105,915 | ||||||||||||||
| Cost of goods sold | 72,929 | 52,943 | 19,986 | |||||||||||||||||
| Gross Profit | 561,281 | 475,352 | 85,929 | |||||||||||||||||
| Operating expenses: | ||||||||||||||||||||
| Research and development | 135,843 | 109,362 | 26,481 | |||||||||||||||||
| Selling, general, and administrative | 383,487 | 323,379 | 60,108 | |||||||||||||||||
| Loss on impairment of assets | 1,702 | — | 1,702 | |||||||||||||||||
| Restructuring charges | — | 9,188 | (9,188) | |||||||||||||||||
| Depreciation and amortization | 7,460 | 8,547 | (1,087) | |||||||||||||||||
| Other (expense) income: | ||||||||||||||||||||
| Interest income | 3,317 | 5,407 | (2,090) | |||||||||||||||||
| Interest expense | (46,159) | (49,598) | 3,439 | |||||||||||||||||
| Other expense | 10,244 | (9,441) | 19,685 | |||||||||||||||||
| Income tax expense | (27,301) | (27,350) | 49 | |||||||||||||||||
| Net loss attributable to common stockholders | $ | (27,110) | $ | (56,106) | $ | 28,996 | ||||||||||||||
Net Product Sales. Net product sales increased $105.9 million during the year ended December 31, 2025 compared to the prior year. The increase was primarily due to both the continued growth of Galafold® and Pombiliti® + Opfolda® in Europe and the U.S. and a $13.5 million favorable impact of foreign currency exchange.
Cost of Goods Sold. Cost of goods sold includes manufacturing costs as well as royalties associated with net product sales. Cost of goods sold increased by $20.0 million primarily related to the increase in net product sales. A portion of Pombiliti® inventory was expensed as research and development costs prior to regulatory approval and as such, the cost of goods sold and related gross margins are not necessarily indicative of future costs of goods sold and gross margin.
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Research and Development Expense. The following table summarizes our principal product development programs for each product candidate in development, and the out-of-pocket, third-party expenses incurred with respect to each product candidate:
| (in thousands) | Years ended December 31, | |||||||||||||
| Projects | 2025 | 2024 | ||||||||||||
| Third-party direct project expenses | ||||||||||||||
Galafold® (Fabry Disease) | $ | 11,229 | $ | 9,298 | ||||||||||
Pombiliti® + Opfolda® (Pompe Disease) | 48,553 | 45,215 | ||||||||||||
| DMX-200 (FSGS kidney disease) | 30,000 | — | ||||||||||||
| Pre-clinical and other programs | 1,518 | 2,437 | ||||||||||||
| Total third-party direct project expenses | 91,300 | 56,950 | ||||||||||||
| Other project costs | 1 | |||||||||||||
| Personnel costs | 34,373 | 41,405 | ||||||||||||
| Other costs | 10,170 | 11,007 | ||||||||||||
| Total other project costs | 44,543 | 52,412 | ||||||||||||
| Total research and development costs | $ | 135,843 | $ | 109,362 | ||||||||||
The $26.5 million increase in research and development costs was primarily driven by the $30.0 million upfront license payment to Dimerix.
Selling, General, and Administrative Expense. Selling, general, and administrative expense increased $60.1 million, primarily driven by higher personnel costs and additional legal and professional fees in connection with our intellectual property litigation and the signing of the Merger Agreement with BioMarin.
Restructuring Charges. Restructuring charges in the prior year were primarily related to an initiative to reduce operating costs by abandoning a lease that we no longer believed was useful in our operations.
Other (Expense) Income. The $19.7 million variance was primarily related to the movement in foreign exchange rates caused by remeasurement of foreign-denominated balances.
Income Tax Expense. The income tax expense for the year ended December 31, 2025 was $27.3 million. We are subject to income taxes in various jurisdictions. Our tax liabilities are largely dependent on the mix of pre-tax earnings among the many jurisdictions in which we operate and differences in the timing of the recognition of such earnings under the relevant accounting standards and tax rules.
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Critical Accounting Policies and Significant Judgments and Estimates
The discussion and analysis of our financial condition and results of operations are based on our financial statements, which we have prepared in accordance with U.S. generally accepted accounting principles ("U.S. GAAP"). The preparation of these financial statements requires us to make estimates and assumptions that affect the reported amounts of assets and liabilities and the disclosure of contingent assets and liabilities at the date of the financial statements, as well as the reported revenues and expenses during the reporting periods. On an ongoing basis, we evaluate our estimates and judgments, including those described in greater detail below. We base our estimates on historical experience and on various other factors that we believe are reasonable under the circumstances, the results of which form the basis for making judgments about the carrying value of assets and liabilities that are not readily apparent from other sources. Actual results may differ from these estimates under different assumptions or conditions. We believe that the following discussion represents our critical accounting policies.
Revenue Recognition
Our net product sales consist of sales of Galafold® for the treatment of Fabry disease and Pombiliti® + Opfolda® for the treatment of Pompe disease. We have recorded revenue on sales where our products are available either on a commercial basis or through a reimbursed early access program. Orders for our products are generally received from distributors and pharmacies with the ultimate payor often a government authority.
We recognize revenue when our performance obligations with our customers have been satisfied, which occurs at a point in time when the pharmacies or distributors obtain control of our products. The transaction price is determined based on fixed consideration in our customer contracts net of estimates for variable consideration. Variable consideration, which primarily consists of discounts and rebates due to foreign and U.S. government programs, is estimated based on contractual arrangements or statutory obligations, which may vary by product and payer and is recorded in the same period the related sales occur.
Estimation requires evaluation of our actual historical experience, customer mix, current contractual and statutory obligations, and inventory channel levels. We evaluate our customer mix to estimate which sales will be subject to which revenue dilutive items and consider changes to government program guidelines or contractual obligations that would impact the actual rebate or discount and/or our estimates of which sales qualify for such rebate or discount. We recognize revenue to the extent that it is probable that a significant revenue reversal will not occur in a future period. These estimates may differ from actual consideration received. We evaluate these estimates each reporting period to reflect known changes.
Liquidity and Capital Resources
As a result of our significant research and development expenditures, as well as expenditures to build a commercial organization to support the launch of Galafold® and Pombiliti® + Opfolda®, we have not been profitable and have generated operating losses since we were incorporated in 2002. We have historically funded our operations through stock offerings, product revenues, debt issuance, collaborations, and other financing arrangements. The Merger Agreement with BioMarin includes restrictions on the conduct of our business prior to the completion of the Merger or termination of the Merger Agreement. These limitations include, among other things, restrictions on our ability to incur additional indebtedness, issue additional shares of our common stock outside of existing equity plans and enter into or amend certain contracts.
Sources of Liquidity
In November 2022, we entered into a Sales Agreement with Goldman Sachs & Co. LLC to create an at-the-market equity program ("ATM program"), pursuant to which we may offer to sell shares of our common stock having aggregate offering gross proceeds of up to $250.0 million. During the year ended December 31, 2025, there were no shares issued and sold through the ATM program. As of December 31, 2025, an aggregate of $164.2 million worth of shares remain available to be issued and sold under the ATM program. Under the Merger Agreement, we are prohibited from offering to sell any additional shares of our common stock under the ATM program.
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In October 2023, we entered into the Senior Secured Term Loan due 2029. This transaction resulted in net proceeds of $387.4 million, after deducting fees and expenses. There were no warrants or equity conversion features associated with the Senior Secured Term Loan due 2029. Simultaneously, we also entered into a securities purchase agreement with funds managed by Blackstone, for the private placement of an aggregate of 2,467,104 shares of our common stock, at a purchase price of $12.16 per share. Proceeds from the private placement, net of offering costs, were $29.8 million. We used proceeds from the Senior Secured Term Loan due 2029 and the private placement to prepay the Senior Secured Term Loan due 2026, inclusive of the outstanding principal amount, accrued interest and prepayment premium.
Cash Flow Discussion
As of December 31, 2025, we had cash, cash equivalents, and marketable securities of $293.5 million. We invest cash in excess of our immediate requirements in regard to liquidity and capital preservation in a variety of interest-bearing instruments, including obligations of U.S. government agencies and money market accounts. Wherever possible, we seek to minimize the potential effects of concentration and degrees of risk. Although we maintain cash balances with financial institutions in excess of insured limits, we do not anticipate any losses with respect to such cash balances. For more details on the cash, cash equivalents, and marketable securities, refer to "— Note 4. Cash, Cash Equivalents, Marketable Securities, and Restricted Cash," in our Notes to Consolidated Financial Statements.
Net Cash Provided by (Used in) Operating Activities
Net cash provided by operations for the year ended December 31, 2025 was $33.1 million. The components of net cash provided by operations primarily reflect net loss of $27.1 million adjusted for non-cash expenses of $99.9 million, which includes $87.4 million of stock compensation and $7.5 million of depreciation expense. This is further driven by a net increase in operating assets and liabilities of $39.6 million. The changes in operating assets and liabilities were primarily driven by an increase in inventory of $111.5 million to support our continued commercial growth, partially offset by an $82.8 million increase in accounts payable and accrued expenses.
Net cash used in operations for the year ended December 31, 2024 was $33.9 million. The components of net cash used in operations included the net loss for the year ended December 31, 2024 of $56.1 million and the net change in operating assets and liabilities of $98.9 million partially offset by $84.9 million of stock compensation and $36.3 million of other non-cash adjustments. The change in operating assets was primarily due to an increase in inventory of $73.7 million to support our continued commercial growth, an increase in accounts receivable of $19.5 million, and a decrease in accounts payable and accrued expenses of $16.5 million associated with timing of payments, partially offset by a decrease in prepaid expenses and other current assets by $14.0 million.
Net Cash Used in Investing Activities
Net cash used in investing activities for the year ended December 31, 2025 was $46.4 million. Our investing activities have consisted primarily of purchases, sales, and maturities of investments and capital expenditures. Net cash used in investing activities reflects $94.4 million for the purchase of marketable securities and $3.3 million for capital expenditures, partially offset by $51.3 million for the sale and redemption of marketable securities.
Net cash used in investing activities for the year ended December 31, 2024 was $0.6 million. Our investing activities have consisted primarily of purchases, sales, and maturities of investments and capital expenditures. Net cash used in investing activities reflects $114.8 million for the purchase of marketable securities and $3.6 million for capital expenditures, partially offset by $117.8 million for the sale and redemption of marketable securities.
Net Cash Provided by Financing Activities
Net cash provided by financing activities for the year ended December 31, 2025 was $0.6 million. Net cash provided by financing activities primarily reflects $19.2 million in proceeds from the exercise of stock options, partially offset by $18.3 million for payments of employee withholding taxes related to restricted stock unit vesting.
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Net cash provided by financing activities for the year ended December 31, 2024 was $5.1 million. Net cash provided by financing activities primarily reflects $19.6 million of net proceeds from the issuance of shares in connection with our ATM program and $7.7 million in proceeds from the exercise of stock options, partially offset by $22.0 million for payments of employee withholding taxes related to restricted stock unit vesting.
Funding Requirements
We expect to continue to incur significant costs in the foreseeable future primarily due to research and development expenses, including expenses related to conducting clinical trials. Our future capital requirements will depend on a number of factors, including:
•the pending transaction with BioMarin;
•the scope, progress, results and costs of clinical trials for our drug candidates;
•the cost of manufacturing drug supply for our commercial, clinical and preclinical studies, including the cost of manufacturing Pombiliti® (also referred to as "ATB200" or "cipaglucosidase alfa");
•the results of preclinical research and clinical trials for DMX-200 and other pipeline candidates we may identify from time to time, including our ability to obtain regulatory approvals and commercialize such therapies;
•the costs, timing, and outcome of regulatory review of our product candidates;
•any changes in regulatory standards relating to the review of our product candidates;
•any changes in laws, rules or regulations, including the imposition of tariffs, most favored nation requirements, or other trade restrictions or requirements, affecting our ability to manufacture, transport, test, develop, or commercialize our products, including Galafold®, Pombiliti® + Opfolda®, or our product candidates;
•the costs of commercialization activities, including product marketing, sales, and distribution;
•the emergence of competing technologies and other adverse market developments;
•the estimates regarding the potential market opportunity for our products and product candidates;
•our ability to successfully commercialize Galafold® (also referred to as "migalastat HCl");
•our ability to successfully commercialize Pombiliti® + Opfolda® in the E.U., U.K., Japan, and U.S., and elsewhere, if regulatory applications are approved;
•our ability to manufacture or supply sufficient clinical trial or commercial products, including DMX-200, Galafold® and Pombiliti® + Opfolda®;
•our ability to obtain reimbursement for Galafold® and Pombiliti® + Opfolda®;
•our ability to satisfy post-marketing commitments or requirements for continued regulatory approval of Galafold® and Pombiliti® + Opfolda®;
•our ability to obtain market acceptance of Galafold® and Pombiliti® + Opfolda® or any other product developed or acquired that has received regulatory approval;
•the costs of preparing, filing, and prosecuting patent applications and maintaining, enforcing, and defending intellectual property-related claims, including Hatch-Waxman litigation;
•the impact of litigation that has been or may be brought against us or of litigation that we are pursuing or may pursue against others, including Hatch-Waxman litigation;
•the extent to which we acquire or invest in businesses, products, and technologies;
•our ability to successfully integrate acquired products and technologies into our business, or successfully divest or license existing products and technologies from our business, including the possibility that the expected benefits of the transactions will not be fully realized by us or may take longer to realize than expected;
•our ability to establish licensing agreements, collaborations, partnerships or other similar arrangements and to obtain milestone, royalty, or other economic benefits from any such collaborators;
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•the costs associated with, and our ability to comply with, emerging sustainability standards, including climate reporting requirements at the local, state and national levels, especially abroad;
•our ability to successfully protect our information technology systems and maintain our global operations and supply chain without interruption;
•our ability to accurately forecast revenue, operating expenditures, or other metrics impacting profitability;
•fluctuations in foreign currency exchange rates; and
•changes in accounting standards.
If the Merger is not consummated, we may seek additional funding through public or private financings of debt or equity. Based on our current operating model and excluding any impact of the pending consummation of the Merger, we believe that the current cash position and expected revenues are sufficient to fund our operations and ongoing research programs for at least the next 12 months. Potential impacts of business development collaborations, pipeline expansion, and investment in manufacturing capabilities could impact our long-term capital requirements.
Contractual Obligations and Commitments
As of December 31, 2025, remaining maturities, including expected interest payments through maturity, on our Senior Secured Term Loan due 2029 were $498.1 million. Refer to "— Note 11. Debt," to the Consolidated Financial Statements for more information.
We are lessees to various operating leases for facilities and equipment. As of December 31, 2025, our undiscounted cash liabilities for operating leases were $72.0 million, with maturities ranging up through fiscal 2034. Refer to “— Note 12. Leases,” to the Consolidated Financial Statements for more information.
On December 19, 2025, the Company entered into the Merger Agreement with BioMarin. Under the terms of the Merger Agreement, the Company may be required to pay BioMarin a termination fee of $175 million if the Merger Agreement is terminated under specific circumstances described in the Merger Agreement.
We have a number of binding minimum purchase and manufacturing commitments due to our third-party manufacturers. As of December 31, 2025, these purchase and manufacturing obligations totaled $218.8 million, of which $195.7 million and $23.1 million are expected in 2026 and 2027, respectively. Contracts for which our commitment is variable, based on volumes, with no fixed minimum quantities, and contracts that can be canceled without payment penalties have been excluded. These purchase obligations are in addition to amounts recorded on our December 31, 2025 Consolidated Balance Sheets.
We have no off-balance sheet arrangements as of December 31, 2025 and 2024.
Recent Accounting Pronouncements
Please refer to "— Note 2. Summary of Significant Accounting Policies," in our Notes to the Consolidated Financial Statements.
Item 7A. QUANTITATIVE AND QUALITATIVE DISCLOSURES ABOUT MARKET RISK
Market risk is the risk of change in fair value of a financial instrument due to changes in interest rates, equity prices, creditworthiness, financing, exchange rates or other factors. Our primary market risk exposure relates to changes in interest rates in our cash, cash equivalents, and marketable securities. We place our investments in high-quality financial instruments, primarily money market funds, asset backed securities, and U.S. government agency notes with maturities of less than one year, which we believe are subject to limited interest rate and credit risk. The securities in our investment portfolio are not leveraged, are classified as available-for-sale and, due to the short-term nature, are subject to minimal interest rate risk. We believe that a 1% (100 basis points) change in average interest rates would either increase or decrease the market value of our investment portfolio by $0.7 million as of December 31, 2025. We currently do not hedge interest rate exposure and consistent with our investment policy, we do not use derivative financial instruments in our investment portfolio.
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We are exposed to interest rate risk with respect to variable rate debt. At December 31, 2025, we had a $400 million Senior Secured Term Loan due 2029 that bears interest at a rate equal to the 3-month Term Secured Overnight Financing Rate ("SOFR"), subject to a 2.5% floor, plus a Term SOFR adjustment of 0.26161% and a margin of 6.25% per year. We entered into this loan in October of 2023, and simultaneously used proceeds from the Senior Secured Term Loan due 2029 and the private placement to prepay the Senior Secured Term Loan due 2026. We do not currently hedge our variable interest rate debt. The annual average variable interest rate for our variable rate debts during the year ended December 31, 2025 was 10.7%. A hypothetical 100 basis point increase or decrease in the average interest rate on our variable rate debts would result in $4.1 million change in the interest expense as of December 31, 2025.
We face foreign exchange risk as a result of entering into transactions denominated in currencies other than U.S. dollars, primarily in British pounds ("GBP") and euros ("EUR"). We are not currently engaged in any foreign currency hedging activities. The current exposures arise primarily from cash, accounts receivable, intercompany receivables and payables, and net product sales denominated in foreign currencies. Both positive and negative impacts to our international product sales from movements in foreign currency exchange rates may be partially mitigated by the natural, opposite impact that foreign currency exchange rates have on our international operating expenses. Based on our foreign currency denominated exposures as of December 31, 2025, we believe that a near-term 10% fluctuation of the U.S. dollar to GBP exchange rate could result in a potential change in the fair value of our net GBP denominated assets and liabilities of approximately $48.6 million. Furthermore, we believe that a near-term 10% fluctuation of the U.S. dollar to EUR exchange rate could result in a potential change in the fair value of our net EUR denominated assets and liabilities of approximately $28.5 million.
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Item 8. FINANCIAL STATEMENTS AND SUPPLEMENTARY DATA
Management's Report on Consolidated Financial Statements and
Internal Control over Financial Reporting
The management of Amicus Therapeutics, Inc. has prepared, and is responsible for the Company's Consolidated Financial Statements and related footnotes. These Consolidated Financial Statements have been prepared in conformity with U.S. generally accepted accounting principles ("U.S. GAAP").
We are responsible for establishing and maintaining adequate internal control over financial reporting. Internal control over financial reporting is defined in Rule 13a-15(f) or 15d-15(f) promulgated under the Securities Exchange Act of 1934 as a process designed by, or under the supervision of the Company's principal executive and principal financial officers and effected by the Company's board of directors, management, and other personnel, to provide reasonable assurance regarding the reliability of financial reporting and the preparation of financial statements for external purposes in accordance with U.S. GAAP and includes those policies and procedures that:
•pertain to the maintenance of records that in reasonable detail accurately and fairly reflect the transactions and dispositions of the assets of Amicus Therapeutics, Inc.;
•provide reasonable assurance that transactions are recorded as necessary to permit preparation of financial statements in accordance with generally accepted accounting principles, and that receipts and expenditures of Amicus therapeutics, Inc. are being made only in accordance with authorizations of management and directors of Amicus therapeutics, Inc.; and
•provide reasonable assurance regarding prevention or timely detection of unauthorized acquisition, use or disposition of the assets of Amicus Therapeutics, Inc. that could have a material effect on the financial statements.
Because of its inherent limitations, internal control over financial reporting may not prevent or detect misstatements. Also, projections of any evaluation of effectiveness to future periods are subject to the risk that controls may become inadequate because of changes in conditions, or that the degree of compliance with the policies or procedures may deteriorate.
We assessed the effectiveness of our internal control over financial reporting as of December 31, 2025. In making this assessment, we used the criteria set forth by the Committee of Sponsoring Organizations of the Treadway Commission (2013 framework) ("COSO") in Internal Control — Integrated Framework. Based on our assessment we believe that, as of December 31, 2025, our internal control over financial reporting is effective based on those criteria.
The effectiveness of the Company's internal control over financial reporting as of December 31, 2025 has been audited by Ernst & Young LLP, an independent registered public accounting firm, as stated in their report. This report appears on the following page.
Dated February 20, 2026
| /s/ BRADLEY L. CAMPBELL | /s/ SIMON HARFORD | |||||||
| President and Chief Executive Officer | Chief Financial Officer | |||||||
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Report of Independent Registered Public Accounting Firm
To the Stockholders and the Board of Directors of Amicus Therapeutics, Inc.
Opinion on Internal Control Over Financial Reporting
We have audited Amicus Therapeutics, Inc.’s internal control over financial reporting as of December 31, 2025, based on criteria established in Internal Control — Integrated Framework issued by the Committee of Sponsoring Organizations of the Treadway Commission (2013 framework) (the COSO criteria). In our opinion, Amicus Therapeutics, Inc. (the Company) maintained, in all material respects, effective internal control over financial reporting as of December 31, 2025, based on the COSO criteria.
We also have audited, in accordance with the standards of the Public Company Accounting Oversight Board (United States) (PCAOB), the consolidated balance sheets of the Company as of December 31, 2025 and 2024, the related consolidated statements of operations, comprehensive loss, changes in stockholders’ equity and cash flows for each of the three years in the period ended December 31, 2025, and the related notes and our report dated February 20, 2026 expressed an unqualified opinion thereon.
Basis for Opinion
The Company’s management is responsible for maintaining effective internal control over financial reporting and for its assessment of the effectiveness of internal control over financial reporting included in the accompanying Management’s Report on Consolidated Financial Statements and Internal Control over Financial Reporting. Our responsibility is to express an opinion on the Company’s internal control over financial reporting based on our audit. We are a public accounting firm registered with the PCAOB and are required to be independent with respect to the Company in accordance with the U.S. federal securities laws and the applicable rules and regulations of the Securities and Exchange Commission and the PCAOB.
We conducted our audit in accordance with the standards of the PCAOB. Those standards require that we plan and perform the audit to obtain reasonable assurance about whether effective internal control over financial reporting was maintained in all material respects.
Our audit included obtaining an understanding of internal control over financial reporting, assessing the risk that a material weakness exists, testing and evaluating the design and operating effectiveness of internal control based on the assessed risk, and performing such other procedures as we considered necessary in the circumstances. We believe that our audit provides a reasonable basis for our opinion.
Definition and Limitations of Internal Control Over Financial Reporting
A company’s internal control over financial reporting is a process designed to provide reasonable assurance regarding the reliability of financial reporting and the preparation of financial statements for external purposes in accordance with generally accepted accounting principles. A company’s internal control over financial reporting includes those policies and procedures that (1) pertain to the maintenance of records that, in reasonable detail, accurately and fairly reflect the transactions and dispositions of the assets of the company; (2) provide reasonable assurance that transactions are recorded as necessary to permit preparation of financial statements in accordance with generally accepted accounting principles, and that receipts and expenditures of the company are being made only in accordance with authorizations of management and directors of the company; and (3) provide reasonable assurance regarding prevention or timely detection of unauthorized acquisition, use, or disposition of the company’s assets that could have a material effect on the financial statements.
Because of its inherent limitations, internal control over financial reporting may not prevent or detect misstatements. Also, projections of any evaluation of effectiveness to future periods are subject to the risk that controls may become inadequate because of changes in conditions, or that the degree of compliance with the policies or procedures may deteriorate.
| /s/ Ernst & Young LLP | ||
Iselin, New Jersey
February 20, 2026
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Report of Independent Registered Public Accounting Firm
To the Stockholders and the Board of Directors of Amicus Therapeutics, Inc.
Opinion on the Financial Statements
We have audited the accompanying consolidated balance sheets of Amicus Therapeutics, Inc. (the Company) as of December 31, 2025 and 2024, the related consolidated statements of operations, comprehensive loss, changes in stockholders’ equity and cash flows for each of the three years in the period ended December 31, 2025, and the related notes (collectively referred to as the "consolidated financial statements"). In our opinion, the consolidated financial statements present fairly, in all material respects, the financial position of the Company at December 31, 2025 and 2024, and the results of its operations and its cash flows for each of the three years in the period ended December 31, 2025, in conformity with U.S. generally accepted accounting principles.
We also have audited, in accordance with the standards of the Public Company Accounting Oversight Board (United States) (PCAOB), the Company’s internal control over financial reporting as of December 31, 2025, based on criteria established in Internal Control — Integrated Framework issued by the Committee of Sponsoring Organizations of the Treadway Commission (2013 framework), and our report dated February 20, 2026 expressed an unqualified opinion thereon.
Basis for Opinion
These financial statements are the responsibility of the Company’s management. Our responsibility is to express an opinion on the Company's financial statements based on our audits. We are a public accounting firm registered with the PCAOB and are required to be independent with respect to the Company in accordance with the U.S. federal securities laws and the applicable rules and regulations of the Securities and Exchange Commission and the PCAOB.
We conducted our audits in accordance with the standards of the PCAOB. Those standards require that we plan and perform the audit to obtain reasonable assurance about whether the financial statements are free of material misstatement, whether due to error or fraud. Our audits included performing procedures to assess the risks of material misstatement of the financial statements, whether due to error or fraud, and performing procedures that respond to those risks. Such procedures included examining, on a test basis, evidence regarding the amounts and disclosures in the financial statements. Our audits also included evaluating the accounting principles used and significant estimates made by management, as well as evaluating the overall presentation of the financial statements. We believe that our audits provide a reasonable basis for our opinion.
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Critical Audit Matter
The critical audit matter communicated below is a matter arising from the current period audit of the financial statements that was communicated or required to be communicated to the audit committee and that: (1) relates to accounts or disclosures that are material to the financial statements and (2) involved our especially challenging, subjective or complex judgments. The communication of the critical audit matter does not alter in any way our opinion on the consolidated financial statements, taken as a whole, and we are not, by communicating the critical audit matter below, providing a separate opinion on the critical audit matter or on the accounts or disclosures to which it relates.
| Revenue recognition – Measurement of variable consideration for Ex-U.S. third-party discounts and rebates | |||||
| Description of the Matter | As described in Note 2 to the consolidated financial statements, the Company recognizes revenue net of estimates for variable consideration, which are primarily third-party discounts and rebates. The sales discounts and rebates are recorded as a reduction of revenue at the time revenue from the sale of the Company’s products is recognized, which occurs at a point in time when the customer obtains control of the product. Auditing the Ex-U.S. sales discounts and rebates was complex because of the volume of sales discounts and rebates and the different contractual product price, discount and/or rebate rate for each country. | ||||
| How We Addressed the Matter in Our Audit | We identified, evaluated, and tested controls over management’s calculations of the discounts and rebates as well as the data input utilized in the calculations. To test the revenue adjustments related to sales discounts and rebates our procedures included, among others, assessing the terms of the arrangement, evaluating the methodology used, testing the significant inputs, and the completeness, accuracy and relevance of the underlying data used by management in its calculations. We inspected significant sales contracts and agreements that include the contractual rights to discounts and rebates and tested credit memos issued during the year and subsequent to year end. In addition, we assessed the historical accuracy of management’s calculations against actual results. | ||||
/s/ | ||
We have served as the Company's auditor since 2003.
February 20, 2026
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Amicus Therapeutics, Inc.
Consolidated Balance Sheets
(in thousands, except share and per share amounts)
| December 31, | |||||||||||
| 2025 | 2024 | ||||||||||
| Assets | |||||||||||
| Current assets: | |||||||||||
| Cash and cash equivalents | $ | $ | |||||||||
| Investments in marketable securities | |||||||||||
| Accounts receivable, net | |||||||||||
| Inventories, net | |||||||||||
| Prepaid expenses and other current assets | |||||||||||
| Total current assets | |||||||||||
| Operating lease right-of-use assets, net | |||||||||||
Property and equipment, less accumulated depreciation of $ | |||||||||||
Intangible assets, less accumulated amortization of $ | |||||||||||
| Goodwill | |||||||||||
| Other non-current assets | |||||||||||
| Total Assets | $ | $ | |||||||||
| Liabilities and Stockholders' Equity | |||||||||||
| Current liabilities: | |||||||||||
| Accounts payable | $ | $ | |||||||||
| Accrued expenses and other current liabilities | |||||||||||
| Operating lease liabilities | |||||||||||
| Total current liabilities | |||||||||||
| Long-term debt | |||||||||||
| Operating lease liabilities | |||||||||||
| Other non-current liabilities | |||||||||||
| Total liabilities | |||||||||||
| Commitments and contingencies | |||||||||||
| Stockholders' equity: | |||||||||||
Common stock, $ | |||||||||||
Common stock in treasury, at cost; | ( | ||||||||||
| Additional paid-in capital | |||||||||||
| Accumulated other comprehensive gain (loss): | |||||||||||
| Foreign currency translation adjustment | |||||||||||
| Unrealized loss on available-for-sale securities | ( | ( | |||||||||
| Warrants | |||||||||||
| Accumulated deficit | ( | ( | |||||||||
| Total stockholders' equity | |||||||||||
| Total Liabilities and Stockholders' Equity | $ | $ | |||||||||
See accompanying Notes to Consolidated Financial Statements
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Amicus Therapeutics, Inc.
Consolidated Statements of Operations
(in thousands, except share and per share amounts)
| Years ended December 31, | |||||||||||||||||
| 2025 | 2024 | 2023 | |||||||||||||||
| Net product sales | $ | $ | $ | ||||||||||||||
| Cost of goods sold | |||||||||||||||||
| Gross profit | |||||||||||||||||
| Operating expenses: | |||||||||||||||||
| Research and development | |||||||||||||||||
| Selling, general, and administrative | |||||||||||||||||
| Changes in fair value of contingent consideration payable | |||||||||||||||||
| Loss on impairment of assets | |||||||||||||||||
| Restructuring charges | |||||||||||||||||
| Depreciation and amortization | |||||||||||||||||
| Total operating expenses | |||||||||||||||||
| Income (loss) from operations | ( | ||||||||||||||||
| Other (expense) income: | |||||||||||||||||
| Interest income | |||||||||||||||||
| Interest expense | ( | ( | ( | ||||||||||||||
| Loss on extinguishment of debt | ( | ||||||||||||||||
Other (expense) income | ( | ( | |||||||||||||||
| Income (loss) before income tax | ( | ( | |||||||||||||||
| Income tax expense | ( | ( | ( | ||||||||||||||
| Net loss attributable to common stockholders | $ | ( | $ | ( | $ | ( | |||||||||||
| Net loss attributable to common stockholders per common share — basic and diluted | $ | ( | $ | ( | $ | ( | |||||||||||
| Weighted-average common shares outstanding — basic and diluted | |||||||||||||||||
See accompanying Notes to Consolidated Financial Statements
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Amicus Therapeutics, Inc.
Consolidated Statements of Comprehensive Loss
(in thousands)
| Years ended December 31, | |||||||||||||||||
| 2025 | 2024 | 2023 | |||||||||||||||
| Net loss | $ | ( | $ | ( | $ | ( | |||||||||||
| Other comprehensive (loss) gain, net of tax: | |||||||||||||||||
| Foreign currency translation adjustment gain (loss) | ( | ||||||||||||||||
| Unrealized gain (loss) on available-for-sale securities | ( | ( | |||||||||||||||
| Other comprehensive gain (loss) | ( | ||||||||||||||||
| Comprehensive loss | $ | ( | $ | ( | $ | ( | |||||||||||
See accompanying Notes to Consolidated Financial Statements
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Amicus Therapeutics, Inc.
Consolidated Statements of Changes in Stockholders' Equity
(in thousands, except share amounts)
| Common Stock | Additional Paid-In Capital | Warrants | Treasury Stock | Other Comprehensive Gain (Loss) | Accumulated Deficit | Total Stockholders' Equity | |||||||||||||||||||||||||||||||||||||||||||||||
| Shares | Amount | Shares | Amount | ||||||||||||||||||||||||||||||||||||||||||||||||||
| Balance at December 31, 2023 | $ | $ | $ | $ | $ | $ | $ | ( | $ | ||||||||||||||||||||||||||||||||||||||||||||
| Stock options exercised, net | — | — | — | — | — | ||||||||||||||||||||||||||||||||||||||||||||||||
| Issuance of shares in connection with at-the-market offering, net of issuance costs | — | — | — | — | — | ||||||||||||||||||||||||||||||||||||||||||||||||
| Vesting of restricted stock units, net of taxes | — | ( | — | — | — | — | — | ( | |||||||||||||||||||||||||||||||||||||||||||||
| Stock-based compensation | — | — | — | — | — | — | — | ||||||||||||||||||||||||||||||||||||||||||||||
| Unrealized loss on available-for-sale securities | — | — | — | — | — | — | ( | — | ( | ||||||||||||||||||||||||||||||||||||||||||||
| Foreign currency translation adjustment | — | — | — | — | — | — | ( | — | ( | ||||||||||||||||||||||||||||||||||||||||||||
| Net loss | — | — | — | — | — | — | — | ( | ( | ||||||||||||||||||||||||||||||||||||||||||||
| Balance at December 31, 2024 | ( | ||||||||||||||||||||||||||||||||||||||||||||||||||||
| Stock options exercised, net | — | — | — | — | — | ||||||||||||||||||||||||||||||||||||||||||||||||
| Vesting of restricted stock units, net of taxes | — | ( | — | — | — | — | — | ( | |||||||||||||||||||||||||||||||||||||||||||||
| Stock-based compensation | — | — | — | — | — | — | — | ||||||||||||||||||||||||||||||||||||||||||||||
| Warrants exercised | — | ( | — | — | — | — | |||||||||||||||||||||||||||||||||||||||||||||||
| Treasury Stock | ( | . | — | ( | — | — | |||||||||||||||||||||||||||||||||||||||||||||||
| Unrealized loss on available-for-sale securities | — | — | — | — | — | — | — | ||||||||||||||||||||||||||||||||||||||||||||||
| Foreign currency translation adjustment | — | — | — | — | — | — | — | ||||||||||||||||||||||||||||||||||||||||||||||
| Net loss | — | — | — | — | — | — | — | ( | ( | ||||||||||||||||||||||||||||||||||||||||||||
| Balance at December 31, 2025 | $ | $ | $ | $ | ( | $ | $ | ( | $ | ||||||||||||||||||||||||||||||||||||||||||||
See accompanying Notes to Consolidated Financial Statements
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Amicus Therapeutics, Inc.
Consolidated Statements of Cash Flows
(in thousands)
| Years ended December 31, | |||||||||||||||||
| 2025 | 2024 | 2023 | |||||||||||||||
| Operating activities | |||||||||||||||||
| Net loss | $ | ( | $ | ( | $ | ( | |||||||||||
| Adjustments to reconcile net loss to net cash used in operating activities: | |||||||||||||||||
| Amortization of debt discount and deferred financing | |||||||||||||||||
| Depreciation and amortization | |||||||||||||||||
| Stock-based compensation | |||||||||||||||||
| Loss on extinguishment of debt | |||||||||||||||||
| Non-cash changes in the fair value of contingent consideration payable | |||||||||||||||||
| Foreign currency remeasurement loss | |||||||||||||||||
| Non-cash deferred taxes | ( | ||||||||||||||||
| Asset impairment charges and other write-offs | ( | ||||||||||||||||
| Changes in operating assets and liabilities: | |||||||||||||||||
| Accounts receivable | ( | ( | ( | ||||||||||||||
| Inventories | ( | ( | ( | ||||||||||||||
| Prepaid expenses and other current assets | ( | ( | |||||||||||||||
| Accounts payable, accrued expenses, and other current liabilities | ( | ||||||||||||||||
| Other non-current assets and liabilities | ( | ( | ( | ||||||||||||||
| Payment of contingent consideration | ( | ||||||||||||||||
| Net cash provided by (used in) operating activities | $ | $ | ( | $ | ( | ||||||||||||
| Investing activities | |||||||||||||||||
| Sale and redemption of marketable securities | |||||||||||||||||
| Purchases of marketable securities | ( | ( | ( | ||||||||||||||
| Capital expenditures | ( | ( | ( | ||||||||||||||
| Net cash (used in) provided by investing activities | $ | ( | $ | ( | $ | ||||||||||||
| Financing activities | |||||||||||||||||
| Proceeds from the issuance of shares in connection with at-the-market offering, net of issuance costs | |||||||||||||||||
| Proceeds from equity financing, net of issuance costs | |||||||||||||||||
| Withholding taxes paid on vested restricted stock units | ( | ( | ( | ||||||||||||||
| Proceeds from stock options exercised, net | |||||||||||||||||
| Proceeds from warrants exercised, net | |||||||||||||||||
| Payment of long-term debt | ( | ||||||||||||||||
| Proceeds from long-term debt, net of issuance costs | |||||||||||||||||
| Payment of contingent consideration | ( | ||||||||||||||||
| Payment of finance leases | ( | ( | ( | ||||||||||||||
| Net cash provided by financing activities | $ | $ | $ | ||||||||||||||
| Effect of exchange rate changes on cash, cash equivalents, and restricted cash | $ | $ | ( | $ | |||||||||||||
| Net increase (decrease) in cash, cash equivalents, and restricted cash | ( | ||||||||||||||||
| Cash, cash equivalents, and restricted cash at the beginning of the year | |||||||||||||||||
| Cash, cash equivalents, and restricted cash at the end of the year | $ | $ | $ | ||||||||||||||
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| Years ended December 31, | |||||||||||||||||
| 2025 | 2024 | 2023 | |||||||||||||||
| Supplemental disclosures of cash flow information | |||||||||||||||||
| Tenant improvements paid through lease incentive | $ | $ | $ | ||||||||||||||
| Cash paid during the period for interest | $ | $ | $ | ||||||||||||||
| Cashless exercise of warrants | $ | $ | $ | ||||||||||||||
| Capital expenditures unpaid at the end of period | $ | $ | $ | ||||||||||||||
| Cash paid for income taxes | $ | $ | $ | ||||||||||||||
See accompanying Notes to Consolidated Financial Statements
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Amicus Therapeutics, Inc.
Notes To Consolidated Financial Statements
1. Description of Business
Amicus Therapeutics, Inc. (the "Company") is a leading, global biotechnology company with a clear and compelling mission to develop and deliver transformative medicines for people living with rare diseases. With extraordinary patient focus, Amicus strives to redefine expectations in rare diseases. The Company's two marketed therapies are Galafold®, the first oral monotherapy for people living with Fabry disease who have amenable genetic variants, and Pombiliti® + Opfolda®, a novel two-component treatment for adults living with late-onset Pompe disease.
Galafold® (also referred to as "migalastat"), is approved in over 40 countries around the world, including the United States ("U.S."), European Union ("E.U."), United Kingdom ("U.K."), and Japan. Additionally, Galafold® has been granted orphan drug designation in the U.S., E.U., U.K., Japan and several other countries.
Pombiliti® + Opfolda® (also referred to as "cipaglucosidase alfa-atga/miglustat"), is approved in the U.S., the E.U., the U.K., Canada, Australia, Switzerland, and Japan. Multiple regulatory submissions and reimbursement processes with global health authorities are currently underway. Additionally, Pombiliti® + Opfolda® has been granted orphan drug designation or status in the U.S., U.K., Switzerland and Japan and data exclusivity in the E.U.
On April 30, 2025, the Company entered into an exclusive license agreement with Dimerix Bioscience Pty Limited ("Dimerix") for the commercialization of Dimerix' Phase 3 drug candidate, DMX-200, in the United States for treatment of Focal Segmental Glomerulosclerosis ("FSGS") and other indications. Refer to "— Note 14. Collaborative Agreements," in the Notes to Consolidated Financial Statements.
On December 19, 2025, the Company entered into a Merger Agreement with BioMarin and its wholly owned acquisition subsidiary. Under the terms of the Merger Agreement, and in accordance with the General Corporation Law of the State of Delaware (the "DGCL"), if the transaction is completed, BioMarin's wholly owned acquisition subsidiary will merge with and into Amicus, and Amicus will continue as the surviving corporation as a wholly owned subsidiary of BioMarin. In addition, all shares of the Company's common stock outstanding immediately prior to closing (other than Excluded Shares or Dissenting Shares (as defined in the Merger Agreement)) will be cancelled and converted into the right to receive $14.50 per share in cash, without interest and subject to any applicable withholding of taxes. Following completion of the Merger, shares of the Company's common stock will no longer be publicly listed. Refer to "— Note 15. Merger Agreement," in the Notes to Consolidated Financial Statements for additional information.
The Company had an accumulated deficit of $2.8 billion as of December 31, 2025. The Company has historically funded its operations through stock offerings, product revenues, debt issuances, collaborations, and other financing arrangements.
Based on its current operating model and excluding any impact of the pending consummation of the Merger, the Company believes the current cash position and expected revenues are sufficient to fund the Company's operations and ongoing research programs for at least the next 12 months. Potential business development opportunities, pipeline expansion, and investment in manufacturing capabilities could impact the Company's long-term capital requirements.
2. Summary of Significant Accounting Policies
Basis of Presentation
The Company has prepared the accompanying Consolidated Financial Statements in accordance with U.S. generally accepted accounting principles ("U.S. GAAP") and include all adjustments necessary for the fair presentation of the Company's financial position for the periods presented. Certain prior year amounts have been reclassified for comparative purposes. The reclassifications did not affect results of operations, net assets or cash flows.
Consolidation
The Consolidated Financial Statements include the accounts of the Company and its subsidiaries. Intercompany accounts and transactions are eliminated in consolidation.
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Amicus Therapeutics, Inc.
Notes To Consolidated Financial Statements — (Continued)
Foreign Currency Transactions
The functional currency for most of the Company's foreign subsidiaries is their local currency. For non-U.S. subsidiaries that transact in a functional currency other than the U.S. dollar, assets and liabilities are translated at current rates of exchange at the balance sheet date. Income and expense items are translated at the weighted average foreign exchange rates for the period. Adjustments resulting from the translation of the financial statements of the Company's foreign operations into U.S. dollars are excluded from the determination of net income and are recorded in accumulated other comprehensive income, a separate component of stockholders' equity. Transactions which are not in the functional currency of the entity are remeasured into the functional currency with gains or losses resulting from the remeasurement recorded in other (expense) income.
Use of Estimates
The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, the disclosure of contingent assets and liabilities at the date of the financial statements, and the reported amounts of revenues and expenses during the reporting periods. Actual results could differ from those estimates.
Cash, Cash Equivalents, Marketable Securities, and Restricted Cash
The Company considers all highly liquid investments purchased with a maturity of three months or less at the date of acquisition to be cash equivalents. Marketable securities consist of fixed income investments with a maturity of greater than three months and other highly liquid investments that can be readily purchased or sold using established markets. These investments are classified as available-for-sale and are reported at fair value on the Company's Consolidated Balance Sheets. Unrealized holding gains and losses are reported within other comprehensive gain (loss) in the Company's Consolidated Statements of Comprehensive Loss. Fair value is based on available market information including quoted market prices, broker or dealer quotations, or other observable inputs.
Concentration of Credit Risk
The Company's financial instruments that are exposed to concentration of credit risk consist primarily of cash, cash equivalents, and marketable securities. The Company maintains its cash and cash equivalents in bank accounts, which, at times, exceed federally insured limits. The Company invests its marketable securities in high-quality commercial financial instruments. The Company has not recognized any losses from credit risks on such accounts during any of the periods presented. The Company believes it is not exposed to significant credit risk on its cash, cash equivalents, or marketable securities.
Property and Equipment
Property and equipment are stated at cost, less accumulated depreciation. Depreciation is calculated over the estimated useful lives of the respective assets, which range from to five years , or the lesser of the related initial term of the lease or useful life for leasehold improvements.
The initial cost of property and equipment consists of its purchase price and any directly attributable costs of bringing the asset to its working condition and location for its intended use. Expenditures incurred after the fixed assets have been put into operation, such as repairs and maintenance, are expensed as incurred. Major replacements, improvements, and additions are capitalized in accordance with Company policy.
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Amicus Therapeutics, Inc.
Notes To Consolidated Financial Statements — (Continued)
The Company evaluates long-lived assets for impairment whenever events or changes in circumstances indicate that the carrying amount of an asset group may not be recoverable. If indications of impairment exist, projected future undiscounted cash flows associated with the asset or asset group are compared to the carrying value of the asset to determine whether the asset or asset group's value is recoverable. If impairment is determined, the Company writes down the asset to its estimated fair value and records an impairment loss equal to the excess of the carrying value of the long-lived asset over its estimated fair value in the period at which such a determination is made.
Revenue Recognition
The Company has recorded revenue on sales where its products are available either on a commercial basis or through a reimbursed early access program. Product orders are generally received from distributors and pharmacies, with the ultimate payor often a government authority. In 2025 and 2024, one customer accounted for 24 % and 26 % of net product sales, respectively. As of December 31, 2025, two customers each accounted for 12 18 % and 13 % of account receivables from product sales, respectively. Revenue attributed to U.K. and Germany accounted for 10 % and 13 % respectively in 2025, each accounted for 13 10 % and 13 % respectively in 2023.
The Company recognizes revenue when its performance obligations to its customers have been satisfied, which occurs at a point in time when the pharmacies or distributors obtain control of the products. The transaction price is determined based on fixed consideration in the Company's customer contracts and is recorded net of estimates for variable consideration, which primarily consist of third-party discounts and rebates. The identified variable consideration is recorded as a reduction of revenue at the time revenue from the sale is recognized. The Company recognizes revenue to the extent that it is probable that a significant revenue reversal will not occur in a future period. These estimates may differ from actual consideration received. The Company evaluates these estimates each reporting period to reflect known changes.
The following table summarizes the Company's net product sales disaggregated by product and geographic area:
| For the Year | ||||||||||||||||||||
| (in thousands) | 2025 | 2024 | 2023 | |||||||||||||||||
Galafold® U.S. | $ | $ | $ | |||||||||||||||||
Galafold® Ex-U.S. | ||||||||||||||||||||
Total Galafold® sales | $ | $ | $ | |||||||||||||||||
Pombiliti® + Opfolda® U.S. | $ | |||||||||||||||||||
Pombiliti® + Opfolda® Ex-U.S. | ||||||||||||||||||||
Total Pombiliti® + Opfolda® sales | $ | $ | $ | |||||||||||||||||
| Total net product sales | $ | $ | $ | |||||||||||||||||
Inventories and Cost of Goods Sold
Until regulatory approval of Pombiliti® + Opfolda®, the Company expensed all manufacturing costs as research and development expense. Upon regulatory approval, the Company began capitalizing costs related to the purchase and manufacture of Pombiliti® + Opfolda®.
Inventories are stated at the lower of cost and net realizable value, determined by the first-in, first-out method. Inventories are reviewed periodically to identify slow-moving or obsolete inventory based on projected sales activity as well as product shelf-life. In evaluating the recoverability of inventories produced, the probability that revenue will be obtained from the future sale of the related inventory is considered and inventory value is written down for inventory quantities in excess of expected requirements. Expired inventory is disposed of and the related costs are recognized as cost of goods sold in the Company's Consolidated Statements of Operations.
Cost of goods sold includes the cost of inventory sold, manufacturing and supply chain costs, product shipping and handling costs, provisions for excess and obsolete inventory, as well as royalties payable. A portion of inventory available for sale was expensed as research and development costs prior to regulatory approval and, as such, the cost of goods sold and related gross margins are not necessarily indicative of future costs of goods sold and gross margin.
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Amicus Therapeutics, Inc.
Notes To Consolidated Financial Statements — (Continued)
Fair Value Measurements
The Company records certain asset and liability balances under the fair value measurements as defined by the Financial Accounting Standard Board ("FASB") guidance. Current FASB fair value guidance emphasizes that fair value is a market-based measurement, not an entity-specific measurement. Therefore, a fair value measurement should be determined based on the assumptions that market participants would use in pricing the asset or liability. As a basis for considering market participant assumptions in fair value measurements, current FASB guidance establishes a fair value hierarchy that distinguishes between market participant assumptions based on market data obtained from sources independent of the reporting entity (observable inputs that are classified within Levels 1 and 2 of the hierarchy) and the reporting entity's own assumptions that market participant's assumptions would use in pricing assets or liabilities (unobservable inputs classified within Level 3 of the hierarchy).
Level 1 inputs utilize quoted prices (unadjusted) in active markets for identical assets or liabilities that the Company has the ability to access at measurement date. Level 2 inputs are inputs other than quoted prices included in Level 1 that are observable for the asset or liability, either directly or indirectly. Level 2 inputs may include quoted prices for similar assets and liabilities in active markets, as well as inputs that are observable for the asset or liability (other than quoted prices), such as interest rates, foreign exchange rates, and yield curves that are observable at commonly quoted intervals. Level 3 inputs are unobservable inputs for the asset or liability, which is typically based on an entity's own assumptions, as there is little, if any, related market activity. In instances where the determination of the fair value measurement is based on inputs from different levels of the fair value hierarchy, the level in the fair value hierarchy within which the entire fair value measurement falls is based on the lowest level input that is significant to the fair value measurement in its entirety. The Company's assessment of the significance of a particular input to the fair value measurement in its entirety requires judgment and considers factors specific to the asset or liability.
Research and Development Costs
Research and development costs are expensed as incurred. Research and development expense consist primarily of costs related to personnel, including salaries and other personnel related expenses, consulting fees, and the cost of facilities and support services used in drug development. Assets acquired that are used for research and development and have no future alternative use are expensed as in-process research and development.
Selling, General and Administrative Expenses
Selling, general and administrative (“SG&A”) expenses relate to sales and marketing, finance, human resources, legal and other administrative activities. SG&A expenses consist primarily of personnel costs, facilities and overhead costs, outside marketing, advertising and legal expenses, and other general and administrative costs.
Interest Income and Interest Expense
Interest income consists of interest earned on the Company's cash, cash equivalents, and marketable securities. Interest expense consists of interest incurred on debt and finance leases.
Income Taxes
The Company accounts for income taxes under the liability method. Under this method deferred income tax liabilities and assets are determined based on the difference between the financial statement carrying amounts and tax basis of assets and liabilities and for operating losses and tax credit carry forwards, using enacted tax rates in effect in the years in which the differences are expected to reverse. A valuation allowance is recorded if it is "more likely than not" that a portion or all of a deferred tax asset will not be realized.
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Amicus Therapeutics, Inc.
Notes To Consolidated Financial Statements — (Continued)
The Company’s tax returns are subject to examination by U.S. Federal, state, and foreign taxing jurisdictions. The impact of an uncertain tax position taken or expected to be taken on an income tax return must be recognized in the financial statements at the largest amount that is more likely than not to be sustained. An uncertain income tax position will not be recognized in the financial statements unless it is more likely than not to be sustained.
Other Comprehensive Gain (Loss)
Components of other comprehensive gain (loss) include unrealized gains and losses on available-for-sale securities and gains and losses on foreign currency transactions, and are included in the Consolidated Statements of Comprehensive Loss.
Leases
The Company primarily enters into lease agreements for office space, equipment, and vehicles. The leases have varying terms, some of which could include options to renew, extend, and early terminate. The Company determines if an arrangement is a lease at contract inception. Operating leases are included in right-of-use ("ROU") assets and lease liabilities on the Consolidated Balance Sheets.
ROU assets represent the Company's right to control the use of an explicitly or implicitly identified fixed asset for a period of time and lease liabilities represent the Company's obligation to make lease payments arising from the lease. Control of an underlying asset is conveyed if the Company obtains the rights to direct the use of and to obtain substantially all of the economic benefits from using the underlying asset. ROU assets and liabilities are recognized at commencement date based on the present value of lease payments over the lease term. The interest rate implicit in lease contracts is typically not readily determinable. As a result, the Company utilizes its incremental borrowing rate, which reflects the fixed rate at which the Company could borrow on a collateralized basis the amount of the lease payments in the same currency, for a similar term, in a similar economic environment.
Lease payments included in the measurement of the lease liability are comprised of fixed payments. Variable lease payments are excluded from the ROU asset and lease liability and are recognized in the period in which the obligation for those payments is incurred. Variable lease payments are presented in the Consolidated Statements of Operations in the same line item as expenses arising from fixed lease payments for operating leases. The Company has lease agreements that include lease and non-lease components, which the Company accounts for as a single lease component for all underlying asset categories.
The lease term for all of the Company's leases includes the non-cancellable period of the lease plus any additional periods covered by either a Company option to extend (or not to terminate) the lease that the Company is reasonably certain to exercise, or an option to extend (or not to terminate) the lease controlled by the lessor.
Leases with an initial term of 12 months or less are not recorded on the Consolidated Balance Sheets. The Company recognizes lease expense for these leases on a straight-line basis over the lease term. The Company applies this policy to all underlying asset categories.
Equity-based Compensation
At December 31, 2025, the Company had one equity-based employee compensation plan, which is described more fully in "— Note 8. Stockholders' Equity." The Company applies the fair value method of measuring equity-based compensation, which requires a public entity to measure the cost of employee services received in exchange for an award of equity instruments based on the grant-date fair value of the award.
Loss per Common Share
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Amicus Therapeutics, Inc.
Notes To Consolidated Financial Statements — (Continued)
Segment Information
The Company currently operates in one one one
Intangible Assets and Goodwill
The Company records goodwill in a business combination when the total consideration exceeds the fair value of the net tangible and identifiable intangible assets acquired. Goodwill is assessed annually for impairment on October 1 and whenever events or circumstances indicate that the carrying amount of an asset may not be recoverable. The Company first assesses the qualitative factors to determine if a quantitative test is necessary. If required, or if the Company elects to bypass the qualitative assessment, a quantitative goodwill impairment test is concluded. If it is determined the Company's single reporting unit's carrying value, including goodwill, exceeds its fair value, an impairment loss is recorded for the difference.
Finite-lived intangible assets are recorded at cost, net of accumulated amortization, and, if applicable, impairment charges. Amortization of finite-lived intangible assets is recorded over the assets’ estimated useful lives on a straight-line basis or based on the pattern in which economic benefits are consumed, if reliably determinable. The Company reviews finite-lived intangible assets for impairment whenever events or changes in circumstances indicate that the carrying amount of an asset may not be recoverable. If impairment is determined, the Company writes down the asset to its estimated fair value and records an impairment loss equal to the excess of the carrying value of the asset over its estimated fair value in the period at which such a determination is made.
No indicators of impairment were noted during the years ended December 31, 2025 and 2024.
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Amicus Therapeutics, Inc.
Notes To Consolidated Financial Statements — (Continued)
Recent Accounting Developments - Guidance Adopted in 2025
In December 2023, the Financial Accounting Standards Board ("FASB") issued Accounting Standards Update ("ASU") No. 2023-09, Income Taxes (Topic 740): Improvements to Income Tax Disclosures. The ASU requires disclosure of disaggregated income taxes paid, prescribes standard categories for the components of the effective tax rate reconciliation, and modifies other income tax-related disclosures. ASU 2023-09 is effective for fiscal years beginning after December 15, 2024, must be applied prospectively with an option to apply retrospectively, and early adoption is permitted. The Company prospectively adopted this guidance on January 1, 2025. This ASU applies to disclosure requirements only, and the Company has included required annual disclosures within "— Note 13. Income Taxes."
Recent Accounting Developments - Guidance Not Yet Adopted
In November 2024, the FASB issued ASU 2024-03, "Income Statement - Reporting Comprehensive Income - Expense Disaggregation Disclosures (Subtopic 220-40): Disaggregation of Income Statement Expenses." The standard is intended to require more detailed disclosures about specified categories of expenses (including employee compensation, depreciation, and amortization) included in certain expense captions presented on the face of the income statement. This ASU is effective for fiscal years beginning after December 15, 2026, and for interim periods within fiscal years beginning after December 15, 2027. Early adoption is permitted. The amendments may be applied either prospectively to financial statements issued for reporting periods after the effective date of this ASU or retrospectively to all prior periods presented in the financial statements. The Company is currently assessing the impact this standard will have on its Consolidated Financial Statements.
In July 2025, the FASB issued ASU 2025-05, "Financial Instruments - Credit Losses (Topic 326)". The standard is intended to simplify the calculation of current expected credit losses by allowing companies to elect a practical expedient to assume that current market conditions as of the balance sheet date will not change over the life of the asset. This ASU is effective for fiscal years beginning after December 15, 2025. Early adoption is permitted. The Company is currently assessing the impact this standard will have on its Consolidated Financial Statements.
In September 2025, the FASB issued ASU 2025-06, "Intangibles-Goodwill and Other-Internal-Use Software (Subtopic 350-40)" related to accounting for internal-use software costs. The amendments in this update improve the operability of the guidance by removing all references to software development project stages so that the guidance is neutral to different software development methods. This update is effective for annual periods beginning after December 15, 2027, including interim periods within those fiscal years, though early adoption is permitted. The Company is currently assessing the impact this standard will have on its Consolidated Financial Statements.
3. Goodwill and Intangible Assets
Finite-lived Intangible Assets
As of December 31, 2025, the Company had intangible assets of $13.9 million. Intangible assets consist of lead enzyme replacement therapy assets acquired with the Callidus Biopharma, Inc. ("Callidus") acquisition in 2013, previously accounted for as in-process research and development. In March 2023, as a result of the European Commission's ("EC") approval of Pombiliti®, the Company began amortizing the assets over the initial regulatory exclusivity period of 7 years. The Company completed an impairment assessment before changing the classification to finite-lived intangible asset noting no impairment. Amortization expense for finite-lived intangible assets was $3.3 million and $3.3 million for the years ended December 31, 2025 and 2024 respectively. Total estimated amortization expense for the finite-lived intangible assets for each of the next 5 years is approximately $3.3 0.8 million in 2030. The finite-lived intangible assets have a remaining amortization period of 4.2 years.
Goodwill
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Amicus Therapeutics, Inc.
Notes To Consolidated Financial Statements — (Continued)
4. Cash, Cash Equivalents, Marketable Securities, and Restricted Cash
As of December 31, 2025, the Company held $214.0 million in cash and cash equivalents and $79.5 million of marketable securities which are reported at fair value on the Company's Consolidated Balance Sheets. Unrealized holding gains and losses are generally reported within other comprehensive gain (loss) in the Consolidated Statements of Comprehensive Loss. If a decline in the fair value of a marketable security below the Company's cost basis is determined to be other-than-temporary or if an available-for-sale debt security’s fair value is determined to be less than the amortized cost and the Company intends or is more than likely to sell the security before recovery and it is not considered a credit loss, such security is written down to its estimated fair value as a new cost basis and the amount of the write-down is included in the Consolidated Statements of Operations as an impairment charge. If the unrealized loss of an available-for-sale debt security is determined to be a result of credit loss the Company would recognize an allowance and the corresponding credit loss would be included in the Consolidated Statements of Operations.
The Company regularly invests excess operating cash in deposits with major financial institutions, money market funds, notes issued by the U.S. government, as well as fixed income investments and U.S. bond funds, both of which can be readily purchased and sold using established markets. The Company believes that the market risk arising from its holdings of these financial instruments is mitigated as, in accordance with Company policy, securities are of high credit rating. Investments that have original maturities greater than three months but less than one year are classified as current. All marketable securities represent the investment of funds available for current operations, notwithstanding their contractual maturities.
Cash, cash equivalents, and marketable securities are classified as current unless mentioned otherwise below and consisted of the following:
| December 31, 2025 | ||||||||||||||||||||||||||
| (in thousands) | Cost | Gross Unrealized Gain | Gross Unrealized Loss | Fair Value | ||||||||||||||||||||||
| Cash and cash equivalents | $ | $ | — | $ | — | $ | ||||||||||||||||||||
| U.S. government agency bonds | ||||||||||||||||||||||||||
| Corporate debt securities | ( | |||||||||||||||||||||||||
| Commercial paper | ( | |||||||||||||||||||||||||
| Money market | ||||||||||||||||||||||||||
| Certificates of deposit | ||||||||||||||||||||||||||
| $ | $ | $ | ( | $ | ||||||||||||||||||||||
| Included in cash and cash equivalents | $ | $ | — | $ | — | $ | ||||||||||||||||||||
| Included in marketable securities | ( | |||||||||||||||||||||||||
| Total cash, cash equivalents, and marketable securities | $ | $ | $ | ( | $ | |||||||||||||||||||||
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Amicus Therapeutics, Inc.
Notes To Consolidated Financial Statements — (Continued)
| December 31, 2024 | ||||||||||||||||||||||||||
| (in thousands) | Cost | Gross Unrealized Gain | Gross Unrealized Loss | Fair Value | ||||||||||||||||||||||
| Cash and cash equivalents | $ | $ | — | $ | — | $ | ||||||||||||||||||||
| Commercial paper | ( | |||||||||||||||||||||||||
| U.S. government agency bonds | ( | |||||||||||||||||||||||||
| Corporate debt securities | ( | |||||||||||||||||||||||||
| Money market | ||||||||||||||||||||||||||
| Certificates of deposit | ||||||||||||||||||||||||||
| $ | $ | $ | ( | $ | ||||||||||||||||||||||
| Included in cash and cash equivalents | $ | $ | — | $ | — | $ | ||||||||||||||||||||
| Included in marketable securities | ( | |||||||||||||||||||||||||
| Total cash, cash equivalents, and marketable securities | $ | $ | $ | ( | $ | |||||||||||||||||||||
For the years ended December 31, 2025 and 2024, there were no
Unrealized loss positions in the marketable securities as of December 31, 2025 reflect temporary impairments and are not a result of credit loss. Additionally, as these positions have been in a loss position for less than twelve months and the Company does not intend to sell these securities before recovery, the losses are recognized in other comprehensive gain (loss). The fair value of these marketable securities in unrealized loss positions are $10.7 million and $23.7 million as of December 31, 2025 and 2024 respectively.
The following table provides a reconciliation of cash, cash equivalents, and restricted cash reported within the Consolidated Balance Sheets that sum to the total of the same such amounts shown in the Consolidated Statements of Cash Flows.
| December 31, | ||||||||||||||||||||
| (in thousands) | 2025 | 2024 | 2023 | |||||||||||||||||
| Cash and cash equivalents | $ | $ | $ | |||||||||||||||||
| Restricted cash | ||||||||||||||||||||
| Cash, cash equivalents, and restricted cash shown in the Consolidated Statements of Cash Flows | $ | $ | $ | |||||||||||||||||
5. Inventories
Inventories consisted of the following:
| December 31, | ||||||||||||||
| (in thousands) | 2025 | 2024 | ||||||||||||
| Raw materials | $ | $ | ||||||||||||
| Work-in-process | ||||||||||||||
| Finished goods | ||||||||||||||
| Total inventories | $ | $ | ||||||||||||
The Company's reserve for inventory was $2.7 million and $3.3 million as of December 31, 2025 and 2024, respectively. The Company has a number of binding minimum purchase and manufacturing commitments due to the third-party manufacturers. As of December 31, 2025, these purchase and manufacturing obligations totaled $218.8 million, of which $195.7 million and $23.1 million are expected in 2026 and 2027, respectively.
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6. Property and Equipment
Property and equipment consist of the following:
| December 31, | ||||||||||||||
| (in thousands) | 2025 | 2024 | ||||||||||||
| Leasehold improvements | $ | $ | ||||||||||||
| Research equipment | ||||||||||||||
| Computer equipment | ||||||||||||||
| Furniture and fixtures | ||||||||||||||
| Computer software | ||||||||||||||
| Construction in progress | ||||||||||||||
| Gross property and equipment | ||||||||||||||
| Less accumulated depreciation | ( | ( | ||||||||||||
| Net property and equipment | $ | $ | ||||||||||||
7. Accrued Expenses and Other Current Liabilities
Accrued expenses and other current liabilities consist of the following:
| December 31, | ||||||||||||||
| (in thousands) | 2025 | 2024 | ||||||||||||
| Accrued sales rebates and discounts | $ | $ | ||||||||||||
| Accrued contract manufacturing & contract research costs | ||||||||||||||
| Accrued compensation and benefits | ||||||||||||||
| Accrued taxes | ||||||||||||||
| Accrued professional fees | ||||||||||||||
| Accrued royalties | ||||||||||||||
| Accrued program fees | ||||||||||||||
| Other | ||||||||||||||
| $ | $ | |||||||||||||
8. Stockholders' Equity
Common Stock and Warrants
As of December 31, 2025, the Company was authorized to issue 500 million shares of common stock. Dividends on common stock will be paid when, and if, declared by the Board of Directors. Each holder of common stock is entitled to vote on all matters that are appropriate for stockholder voting and is entitled to one vote for each share held.
In November 2022, the Company entered into a Sales Agreement with Goldman Sachs & Co. LLC to create an at-the-market equity program ("ATM program"), pursuant to which the Company may offer to sell shares of its common stock having an aggregate offering gross proceeds of up to $250.0 million. There were no shares issued and sold during the year ended December 31, 2025 through the ATM program. During the year ended December 31, 2024, the Company issued and sold an aggregate of 1,732,204 shares through its ATM program at a weighted-average public offering price of $11.69 per share, resulting in net proceeds of $19.6 million. As of December 31, 2025, an aggregate of $164.2 million worth of shares remain available to be issued and sold under the ATM program.
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In October 2023, the Company entered into a securities purchase agreement with funds managed by Blackstone Alternative Credit Advisors LP and Blackstone Life Sciences Advisors L.L.C. (collectively, “Blackstone”), for the private placement of an aggregate of 2,467,104 shares of the Company’s common stock, at a purchase price of $12.16 per share. Proceeds from the private placement, net of offering costs, were $29.8 million.
Equity Incentive Plan
The Company's stockholders approved the 2025 Equity Incentive Plan (the "2025 Equity Plan") at the 2025 Annual Meeting of Stockholders, held on June 5, 2025 (the “2025 Annual Meeting”), to replace the expiring Amended and Restated 2007 Equity Incentive Plan (the "2007 Equity Plan"). Following approval of the 2025 Equity Plan, no future awards are permitted to be granted under the 2007 Equity Plan. The maximum number of shares of our common stock that may be issued under the 2025 Equity Plan (subject to certain adjustments) is the sum of (i) 9.0 million shares which were approved by stockholders at the 2025 Annual Meeting; plus (ii) the number of shares reserved for issuance under the 2007 Equity Plan that remained available for grant under the 2007 Equity Plan as of the 2025 Annual Meeting; plus (iii) any shares underlying 2007 Equity Plan awards that may become available for issuance under the 2025 Equity Plan in accordance with the 2025 Equity Plan provisions.
The 2025 Equity Plan provides for the granting of restricted stock units and options to purchase common stock in the Company to employees, directors, advisors, and consultants at a price to be determined by the Company's Board of Directors. The 2025 Equity Plan is intended to encourage ownership of stock by employees and consultants of the Company and to provide additional incentives for them to promote the success of the Company's business. Under the provisions of the 2025 Equity Plan, no option will have a term in excess of 10 years. The Board of Directors, or its committee, is responsible for determining the individuals to be granted options, the number of options each individual will receive, the option price per share, and the exercise period of each option. Options granted pursuant to the 2025 Equity Plan generally vest 25 % on the first year anniversary date of grant plus an additional 1/48th for each month thereafter and may be exercised in whole or in part for 100 % of the shares vested at any time after the date of grant. As of December 31, 2025, the Company has reserved up to 15,294,241 shares for issuance under the 2025 Equity Plan.
9. Stock-based Compensation
The Plan provides for the granting of restricted stock units and options to purchase common stock in the Company to employees, directors, advisors, and consultants at a price to be determined by the Company’s Board of Directors. The Plan is intended to encourage ownership of stock by employees and consultants of the Company and to provide additional incentives for them to promote the success of the Company’s business. The Board of Directors, or its committee, is responsible for determining the individuals to be granted options, the number of options each individual will receive, the option price per share, and the exercise period of each option.
The Plan provides for certain benefits to qualifying Plan participants who separate from service with the Company due to death, disability or "retirement" (as such term is defined under the Plan) ("Qualified Participants"). Options granted under the Plan to a Qualified Participant shall continue to vest until the 2 nd anniversary of the Qualified Participant’s separation and all vested options held by such Qualified Participant shall remain exercisable until the earlier of the 4 th anniversary of the Qualified Participant’s separation or the original expiration date of the option. Options that are not exercised during this exercise period shall be forfeited. Time-based restricted stock units and restricted stock granted to a Qualified Participant under the Plan that was scheduled to vest within the two-year period following the Qualified Participant's separation shall accelerate and be delivered upon such separation. Any time-based restricted stock units or restricted stock that would have vested after such two-year period will be forfeited upon the Qualified Participant's separation. Also, per the Amendment, any performance-based restricted stock units under the Plan ("PRSUs") received by the Qualified Participant, shall remain eligible to vest after the Qualified Participant’s separation based on the actual performance of the Company through the end of the performance period applicable to any such PRSUs.
Stock Option Grants
The Company uses the fair value method of measuring share-based compensation, using the fair value of each equity award granted. The Company chose the ‘‘straight-line’’ attribution method for allocating compensation costs and recognized the fair value of each stock option on a straight-line basis over the vesting period of the related awards.
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The Company uses the Black-Scholes option pricing model when estimating the grant date fair value for share-based awards. Use of a valuation model requires management to make certain assumptions with respect to selected model inputs. Expected volatility is based on the historical volatility of the Company's common stock over the look-back period corresponding to the expected life of the options. The average expected life is determined using the Company's actual historical data. The risk-free interest rate is based on U.S. Treasury, zero-coupon issues with a remaining term equal to the expected life assumed at the date of grant. Forfeitures are estimated on the grant date and reassessed each reporting period based on historical experience and management's expectations of future forfeitures. To the extent actual forfeitures differ from the estimates, the difference is recorded as a cumulative adjustment in the period in which the estimates are revised.
The fair value of the stock options granted were estimated on the date of grant using a Black-Scholes option pricing model with the following weighted-average assumptions:
| Years ended December 31, | ||||||||||||||||||||
| 2025 | 2024 | 2023 | ||||||||||||||||||
| Expected stock price volatility | % | % | % | |||||||||||||||||
| Risk free interest rate | % | % | % | |||||||||||||||||
| Expected life of options (years) | ||||||||||||||||||||
| Expected annual dividend per share | $ | $ | $ | |||||||||||||||||
The weighted average grant-date fair value per share of options granted during 2025, 2024, and 2023 were $4.68 , $7.50 , and $6.75 , respectively.
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A summary of the Company's stock options is as follows:
| Number of Shares | Weighted Average Exercise Price | Weighted Average Remaining Years | Aggregate Intrinsic Value | ||||||||||||||||||||
| (in thousands) | (in millions) | ||||||||||||||||||||||
| Options outstanding, December 31, 2022 | $ | ||||||||||||||||||||||
| Granted | $ | ||||||||||||||||||||||
| Exercised | ( | $ | |||||||||||||||||||||
| Forfeited | ( | $ | |||||||||||||||||||||
| Expired | ( | $ | |||||||||||||||||||||
| Options outstanding, December 31, 2023 | $ | ||||||||||||||||||||||
| Granted | $ | ||||||||||||||||||||||
| Exercised | ( | $ | |||||||||||||||||||||
| Forfeited | ( | $ | |||||||||||||||||||||
| Expired | ( | $ | |||||||||||||||||||||
| Options outstanding, December 31, 2024 | $ | ||||||||||||||||||||||
| Granted | $ | ||||||||||||||||||||||
| Exercised | ( | $ | |||||||||||||||||||||
| Forfeited | ( | $ | |||||||||||||||||||||
| Expired | ( | $ | |||||||||||||||||||||
| Options outstanding, December 31, 2025 | $ | $ | |||||||||||||||||||||
| Vested and unvested expected to vest, December 31, 2025 | $ | $ | |||||||||||||||||||||
| Exercisable at December 31, 2025 | $ | $ | |||||||||||||||||||||
The aggregate intrinsic value of options exercised during the years ended December 31, 2025, 2024 and 2023 was $6.6 million, $3.8 million, and $7.1 million, respectively. Cash proceeds from stock options exercised during the years ended December 31, 2025, 2024, and 2023 were $19.2 million, $7.7 million, and $10.3 million, respectively. As of December 31, 2025, the total unrecognized compensation cost related to non-vested stock options granted was $33.6 million and is expected to be recognized over a weighted average period of two years .
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Restricted Stock Units and Performance-Based Restricted Stock Units (collectively "RSUs")
RSUs awarded under the Plan are generally subject to graded vesting and are contingent on an employee's continued service. RSUs are generally subject to forfeiture if employment terminates prior to the release of vesting restrictions. The Company expenses the cost of the RSUs, which is determined to be the fair market value of the shares of common stock underlying the RSUs at the date of grant, ratably over the period during which the vesting restrictions lapse. A summary of non-vested RSU activity under the Plan is as follows:
| Number of Shares | Weighted Average Grant Date Fair Value | Weighted Average Remaining Years | Aggregate Intrinsic Value | ||||||||||||||||||||
(in thousands) | (in millions) | ||||||||||||||||||||||
| Non-vested units as of December 31, 2022 | $ | ||||||||||||||||||||||
| Granted | $ | ||||||||||||||||||||||
| Vested | ( | $ | |||||||||||||||||||||
| Forfeited | ( | $ | |||||||||||||||||||||
| Non-vested units as of December 31, 2023 | $ | ||||||||||||||||||||||
| Granted | $ | ||||||||||||||||||||||
| Vested | ( | $ | |||||||||||||||||||||
| Forfeited | ( | $ | |||||||||||||||||||||
| Non-vested units as of December 31, 2024 | $ | ||||||||||||||||||||||
| Granted | $ | ||||||||||||||||||||||
| Vested | ( | $ | |||||||||||||||||||||
| Forfeited | ( | $ | |||||||||||||||||||||
| Non-vested units as of December 31, 2025 | $ | $ | |||||||||||||||||||||
As of December 31, 2025, there was $72.3 million of total unrecognized compensation cost related to unvested RSUs with service-based vesting conditions. These costs are expected to be recognized over a weighted average period of two years .
Compensation Expense Related to Equity Awards
The following table summarizes information related to compensation expense recognized in the Company's Consolidated Statements of Operations related to the equity awards:
| Years ended December 31, | ||||||||||||||||||||
| (in thousands) | 2025 | 2024 | 2023 | |||||||||||||||||
| Research and development expense | $ | $ | $ | |||||||||||||||||
| Selling, general, and administrative expense | ||||||||||||||||||||
| Total equity compensation expense | $ | $ | $ | |||||||||||||||||
10. Assets and Liabilities Measured at Fair Value
The Company's financial assets and liabilities are measured at fair value and classified within the fair value hierarchy which is defined as follows:
Level 1 — Quoted prices in active markets for identical assets or liabilities that the Company has the ability to access at the measurement date.
Level 2 — Inputs other than quoted prices in active markets that are observable for the asset or liability, either directly or indirectly.
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Level 3 — Inputs that are unobservable for the asset or liability.
A summary of the fair value of the Company's recurring assets and liabilities aggregated by the level in the fair value hierarchy within which those measurements fall as of December 31, 2025 are identified in the following tables:
| (in thousands) | Level 1 | Level 2 | Total | |||||||||||||||||
| Assets: | ||||||||||||||||||||
| U.S. government agency bonds | $ | $ | $ | |||||||||||||||||
| Corporate debt securities | ||||||||||||||||||||
| Commercial paper | ||||||||||||||||||||
| Money market | ||||||||||||||||||||
| $ | $ | $ | ||||||||||||||||||
| (in thousands) | Level 1 | Level 2 | Total | |||||||||||||||||
| Liabilities: | ||||||||||||||||||||
| Deferred compensation plan liability | $ | $ | $ | |||||||||||||||||
| $ | $ | $ | ||||||||||||||||||
A summary of the fair value of the Company's recurring assets and liabilities aggregated by the level in the fair value hierarchy within which those measurements fall as of December 31, 2024 are identified in the following tables:
| (in thousands) | Level 1 | Level 2 | Total | |||||||||||||||||
| Assets: | ||||||||||||||||||||
| Commercial paper | $ | $ | $ | |||||||||||||||||
| U.S. government agency bonds | ||||||||||||||||||||
| Corporate debt securities | ||||||||||||||||||||
| Money market | ||||||||||||||||||||
| $ | $ | $ | ||||||||||||||||||
| (in thousands) | Level 1 | Level 2 | Total | |||||||||||||||||
| Liabilities: | ||||||||||||||||||||
| Deferred compensation plan liability | ||||||||||||||||||||
| $ | $ | $ | ||||||||||||||||||
The Company's Senior Secured Term Loan due 2029 falls into the Level 2 category within the fair value level hierarchy and the fair value was determined using a discounted cash flow analysis that factors in current market yields for comparable borrowing arrangements under the Company's credit profile. The carrying value of the Senior Secured Term Loan due 2029 approximates the fair value. Deferred compensation plan liability is recorded as a component of other non-current liabilities on the Company's Consolidated Balance Sheets.
The Company did not have any Level 3 assets or liabilities as of December 31, 2025 or 2024.
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Cash, Money Market Funds and Marketable Securities
The Company classifies its cash and money market funds within the fair value hierarchy as Level 1 as these assets are valued using quoted prices in an active market for identical assets at the measurement date. The Company considers its investments in marketable securities as available-for-sale and classifies these assets within the fair value hierarchy as Level 2 primarily utilizing broker quotes in a non-active market for valuation of these securities. No changes in valuation techniques or inputs occurred during the year ended December 31, 2025. No transfers of assets between Level 1 and Level 2 of the fair value measurement hierarchy occurred during the year ended December 31, 2025.
11. Debt
The Company's debt consists of the following:
| December 31, | ||||||||||||||
| (in thousands) | 2025 | 2024 | ||||||||||||
Senior Secured Term Loan due 2029: | ||||||||||||||
| Principal | $ | $ | ||||||||||||
Less: debt discount (1) | ( | ( | ||||||||||||
Less: deferred financing (1) | ( | ( | ||||||||||||
| Net carrying value of long-term debt | $ | $ | ||||||||||||
_____________________________
(1) Included in the Consolidated Balance Sheets within long-term debt and amortized to interest expense over the remaining life of the corresponding Senior Secured Term Loan using the effective interest rate method.
Senior Secured Term Loan due 2029
In October 2023, the Company entered into a $400 million loan agreement (the “Senior Secured Term Loan due 2029”) with Blackstone Alternative Credit Advisors LP and Blackstone Life Sciences Advisors L.L.C. (collectively, “Blackstone”) with an interest rate equal to 3-month Term SOFR, subject to a 2.5 % floor, plus a Term SOFR adjustment of 0.26161 % and a margin of 6.25 % that requires interest-only payments until early-2027. The Senior Secured Term Loan due 2029 will be repaid in twelve quarterly payments of $33.3 million, starting on January 2027 with the final balance due on the maturity date in October 2029. This transaction resulted in net proceeds of $387.4 million, after deducting fees and expenses. There were no warrants or equity conversion features associated with the Senior Secured Term Loan due 2029.
The Senior Secured Term Loan due 2029 is subject to mandatory prepayment provisions that require prepayment upon a change of control, the incurrence of certain additional indebtedness, asset sale, or an event of loss, subject to certain conditions set forth in the Senior Secured Term Loan due 2029. The Company may prepay the Senior Secured Term Loan due 2029 in whole or in part, at its option at any time. Any prepayment of the Senior Secured Term Loan due 2029 is subject to certain make-whole premiums and prepayment premiums, the latter of which decrease until the fifth anniversary of the transaction date at which point no prepayment penalty shall exist. The obligations under the Senior Secured Term Loan due 2029 are secured by a first lien security interest in certain assets of the Company. The Senior Secured Term Loan due 2029 contains certain customary representations and warranties, affirmative and negative covenants and events of default applicable to the Company. If an event of default occurs and is continuing, Blackstone may declare all amounts outstanding under the Senior Secured Term Loan due 2029 to be immediately due and payable.
Senior Secured Term Loan due 2026
In July 2020, the Company entered into a definitive agreement for a $400 million credit facility with Hayfin Capital Management (“Senior Secured Term Loan due 2026”) with an interest rate equal to 3-month LIBOR, subject to a 1 % floor, plus 6.5 % per annum and requires interest-only payments until mid-2024. The Senior Secured Term Loan due 2026 was to be repaid in nine quarterly payments of $44.4 million, starting on July 2024 with the final balance due on the maturity date in July 2026. There were no warrants or equity conversion features associated with the Senior Secured Term Loan due 2026.
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In October 2023, the Company used $408.9 million of the net proceeds from the Senior Secured Term Loan due 2029 and the private placement to prepay the Senior Secured Term Loan due 2026, inclusive of the outstanding principal amount, $0.8 million in accrued interest and $8.0 million as a prepayment premium. In connection with the prepayment, the Company recorded a loss from early extinguishment of debt of $13.9 million in the Company's Consolidated Statements of Operations for the year ended December 31, 2023.
Interest Expense
The following table sets forth interest expense recognized related to the Company's debt for the years ended December 31, 2025 and 2024, respectively:
| December 31, | ||||||||||||||
| (in thousands, except interest rate amounts) | 2025 | 2024 | ||||||||||||
| Contractual interest expense | $ | $ | ||||||||||||
| Amortization of debt discount | $ | $ | ||||||||||||
| Amortization of deferred financing | $ | $ | ||||||||||||
| Effective interest rate of the liability component, Senior Secured Term Loan due 2029 | % | % | ||||||||||||
12. Leases
The Company currently has operating leases for office and research laboratory space, equipment, and vehicles under agreements expiring at various dates through 2034, which include renewal options on leases which the Company is reasonably certain to exercise.
For the years ended December 31, 2025 and 2024, operating lease expense was $6.7 million and $6.7 million and variable lease expense was $2.0 million and $2.2 million, respectively. For the years ended December 31, 2025 and 2024, the Company paid $9.4 million and $9.6 million, respectively, for amounts related to our operating lease liabilities and recorded right-of-use assets of $0.3 million and $0.7 million, respectively.
Commitments under finance leases are not significant for the year ended December 31, 2025.
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Supplemental balance sheet information related to operating leases were as follows:
| December 31, | ||||||||||||||
| (in thousands, except year and discount rate amounts) | 2025 | 2024 | ||||||||||||
| Operating lease ROU assets, net | $ | $ | ||||||||||||
| Current portion of the operating lease liabilities | $ | $ | ||||||||||||
| Non-current portion of the operating lease liabilities | $ | $ | ||||||||||||
| Total operating lease liability | $ | $ | ||||||||||||
| Weighted-average remaining lease terms (years) | ||||||||||||||
| Weighted-average discount rate | % | % | ||||||||||||
At December 31, 2025, the future minimum operating lease payments were as follows:
| (in thousands) | Operating Lease | |||||||
| 2026 | $ | |||||||
| 2027 | ||||||||
| 2028 | ||||||||
| 2029 | ||||||||
| 2030 | ||||||||
| Thereafter | ||||||||
| Total lease payments | ||||||||
| Less: lease incentives | ( | |||||||
| Less: imputed interest | ( | |||||||
| Total operating lease liability | $ | |||||||
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13. Income Taxes
For financial reporting purposes, income (loss) before income taxes includes the following components:
| Years ended December 31, | ||||||||||||||||||||
| (in thousands) | 2025 | 2024 | 2023 | |||||||||||||||||
| United States | $ | ( | $ | ( | $ | ( | ||||||||||||||
| Foreign | ||||||||||||||||||||
| Total | $ | $ | ( | $ | ( | |||||||||||||||
Following were the components of income tax expense (benefit) for the years ended December 31, 2025, 2024, and 2023:
| Years ended December 31, | ||||||||||||||||||||
| (in thousands) | 2025 | 2024 | 2023 | |||||||||||||||||
| Current | ||||||||||||||||||||
| Federal | $ | $ | $ | |||||||||||||||||
| State | ||||||||||||||||||||
| Foreign | ||||||||||||||||||||
| Deferred | ||||||||||||||||||||
| Federal | ( | |||||||||||||||||||
| State | ( | |||||||||||||||||||
| Foreign | ||||||||||||||||||||
| Total | $ | $ | $ | |||||||||||||||||
A reconciliation of the statutory tax rates and the effective tax rates for the year ended December 31, 2025 is as follows:
| Year ended December 31, 2025 | ||||||||||||||
| (in thousands) | Amount | Percent | ||||||||||||
| U.S. Federal statutory tax rate | $ | % | ||||||||||||
| Foreign tax effects | ||||||||||||||
| United Kingdom | ||||||||||||||
| Statutory rate difference between the United Kingdom and the United States | % | |||||||||||||
| Nontaxable or nondeductible items | ||||||||||||||
| Intercompany sales income | ( | ( | % | |||||||||||
| Executive compensation | % | |||||||||||||
| Other | ( | ( | % | |||||||||||
| Change in valuation allowance | ( | ( | % | |||||||||||
| Other adjustments | ||||||||||||||
| Deferred charge | ( | ( | % | |||||||||||
| Return to provision | ( | ( | % | |||||||||||
| Ireland | ||||||||||||||
| Statutory rate difference between Ireland and the United States | ( | ( | % | |||||||||||
| Nontaxable or nondeductible items | ||||||||||||||
| Intercompany sales income | ( | ( | % | |||||||||||
| Meals and entertainment | % | |||||||||||||
| Other adjustments | ||||||||||||||
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| Return to provision | % | |||||||||||||
| Other | % | |||||||||||||
| Japan | ||||||||||||||
| Other adjustments | ||||||||||||||
| Return to provision | ( | ( | % | |||||||||||
| Other | ( | ( | % | |||||||||||
| Germany | ||||||||||||||
| Statutory rate difference between Germany and the United States | % | |||||||||||||
| Other | % | |||||||||||||
| Other foreign jurisdictions | % | |||||||||||||
| Effects of changes in tax laws or rates enacted in the current period | % | |||||||||||||
| Effect of cross-border tax laws | ||||||||||||||
| Subpart F | % | |||||||||||||
| Global intangible low taxed income | % | |||||||||||||
| Tax credits | ||||||||||||||
| Research and development tax credits | ( | ( | % | |||||||||||
| Changes in valuation allowance | % | |||||||||||||
| Nontaxable or nondeductible items | ||||||||||||||
| Executive compensation | % | |||||||||||||
| 162(m) limitation | % | |||||||||||||
| Intercompany royalty | % | |||||||||||||
| Other | % | |||||||||||||
| Changes in unrecognized tax benefits | % | |||||||||||||
| Other adjustments | ||||||||||||||
| Unrealized FX gain/loss | ( | ( | % | |||||||||||
| 986c FX gain/loss | ( | ( | % | |||||||||||
| Foreign withholding tax | % | |||||||||||||
| Return to provision | ( | ( | % | |||||||||||
| Other | % | |||||||||||||
| Total effective tax rate | $ | % | ||||||||||||
A reconciliation of the statutory tax rates and the effective tax rates for the years ended December 31, 2024 and 2023 are as follows:
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Years ended December 31, | ||||||||||||||
2024 | 2023 | |||||||||||||
Statutory rate | ( | % | ( | % | ||||||||||
Tax credits | ( | % | ( | % | ||||||||||
Impact of foreign operations | % | ( | % | |||||||||||
Executive compensation | % | % | ||||||||||||
Other | % | % | ||||||||||||
Valuation allowance | ( | % | % | |||||||||||
Net | % | % | ||||||||||||
The amounts of cash taxes paid by the Company is as follows:
| (in thousands) | Year ended December 31, 2025 | |||||||
| Federal | $ | |||||||
| State | ||||||||
| Foreign: | ||||||||
| United Kingdom | ||||||||
| Ireland | ||||||||
| Germany | ||||||||
| All other foreign | ||||||||
| Total | $ | |||||||
On December 22, 2017, the U.S. government enacted the Tax Cuts and Jobs Act ("Tax Act"). The Tax Act significantly revised U.S. tax law by, among other provisions, lowering the U.S. federal statutory income tax rate to 21%, imposing a mandatory one-time transition tax on previously deferred foreign earnings, and eliminating or reducing certain income tax deductions. The Tax Act also introduced an additional U.S. tax on certain non-U.S. subsidiaries’ earnings which are considered to be Global Intangible Low Taxed Income (referred to as "GILTI"). After consideration of the relevant guidance and completing the accounting for the tax effects of the Tax Act, the Company has elected to treat GILTI as a period cost.
Beginning in 2022, the Tax Act eliminated the right to deduct research and development expenditures for tax purposes in the period the expenses were incurred and instead requires all U.S. and foreign research and development expenditures to be amortized over five and fifteen tax years, respectively.
On July 4, 2025, the U.S. government enacted the budget reconciliation bill, H.R. 1, also referred to as, the One Big Beautiful Bill Act of 2025 (“OBBBA”). The OBBBA contains several changes to corporate taxation including modifications to capitalization of research and development expenses, limitations on deductions for interest expense and accelerated fixed asset depreciation. The legislation has multiple effective dates, with certain provisions effective in 2025 and others implemented through 2027. The changes resulting from the tax provisions in OBBBA are not expected to have a material impact on the Company’s results of operations.
Tax returns for years 2021 through 2025 are open to examination by tax authorities. The Company is also subject to examination in any period for which it has net operating losses. A reconciliation of the beginning and ending amount of unrecognized tax benefits is as follows:
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| Years ended December 31, | ||||||||||||||||||||
| (in thousands) | 2025 | 2024 | 2023 | |||||||||||||||||
| Gross unrecognized tax benefits at beginning of year | $ | $ | $ | |||||||||||||||||
| Gross increases for tax positions in prior years | ||||||||||||||||||||
| Decreases for settlements and payments | ( | |||||||||||||||||||
| Gross unrecognized tax benefits at end of year | $ | $ | $ | |||||||||||||||||
For the years ended December 31, 2025 and 2024, approximately $21.3 million and $13.8 million of unrecognized tax benefits respectively, would, if recognized, impact the Company's effective tax rate. The Company recognizes accrued interest and penalties related to its uncertain tax positions as part of its income tax expense within its consolidated statement of operations. The amount of accrued interest and penalties was $5.7 million for the year ended December 31, 2025.
Deferred income taxes reflect the net effect of temporary difference between the carrying amounts of assets and liabilities for financial reporting purposes and the amounts used for income tax purposes. The significant components of the deferred tax assets and liabilities are as follows:
| Years ended December 31, | ||||||||||||||
| (in thousands) | 2025 | 2024 | ||||||||||||
| Deferred tax assets | ||||||||||||||
| Net operating loss carry forwards | $ | $ | ||||||||||||
| Research tax credit | ||||||||||||||
| Stock-based compensation | ||||||||||||||
| Capitalized research and development costs | ||||||||||||||
| Interest carry forward limitation | ||||||||||||||
| Lease liability | ||||||||||||||
| Inventory | ||||||||||||||
| Other | ||||||||||||||
| Gross deferred tax assets | ||||||||||||||
| Deferred tax liabilities | ||||||||||||||
| Other | ( | ( | ||||||||||||
| Total net deferred tax assets | ||||||||||||||
| Less: valuation allowance | ( | ( | ||||||||||||
| Net deferred tax liability | $ | $ | ||||||||||||
The Company records a valuation allowance for temporary differences for which it is more likely than not that the Company will not receive future tax benefits. At December 31, 2025 and 2024, the Company recorded valuation allowances of $622.1 million and $609.1 million, respectively, representing an increase in the valuation allowance of $13.0 million in 2025, due to the uncertainty regarding the realization of such deferred tax assets, to offset the benefits of net operating losses generated during those years. The deferred tax liability related to business acquisitions pertains to the basis difference in intangible assets acquired by the Company. The Company's policy is to record a deferred tax liability related to acquired intangible assets that may eventually be realized either upon amortization of the asset when the research is completed, and a product is successfully launched or the write-off of the asset if it is abandoned or unsuccessful.
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As of December 31, 2025, the Company had U.S. federal and state net operating loss carry forwards ("NOLs") of approximately $1.2 billion and $1.0 billion, respectively. The federal carry forward for losses generated prior to 2018 will expire in 2033 through 2037. Federal net operating losses incurred in 2018 and onward have an indefinite expiration under the Tax Act. Most of the state net operating loss carry forwards generated prior to 2009 have expired through 2016. The remaining state net operating loss carry forwards including those generated in 2009 through 2025 will expire in 2028 through 2045. State research and development credits will expire 2026 through 2033. Utilization of NOLs may be subject to a substantial limitation pursuant to Section 382 of the Internal Revenue Code of 1986, as amended ("Section 382"), as well as similar state statutes in the event of an ownership change. Such ownership changes have occurred in the past and could occur again in the future. Under Section 382, if a corporation undergoes an "ownership change," generally defined as a greater than 50% change (by value) in its equity ownership over a three-year period, the corporation's ability to use its pre-change NOLs and other pre-change tax attributes (such as research and development tax credits) to offset its post-change income may be limited. The Company completed a detailed study of the NOLs for the tax year 2024 and determined that there was not an ownership change in excess of 50%. The Company may experience ownership changes in the future as a result of shifts in the stock ownership some of which are outside the Company's control. Ownership changes in future periods may place additional limits on the Company's ability to utilize net operating loss and tax credit carry forwards. In addition, at the state level, there may be periods during which the use of NOLs is suspended or otherwise limited, which could accelerate or permanently decrease the amount of state attributes and increase state taxes owed.
The Company also has U.S. federal research and experimentation and orphan drug credit carryforwards of approximately $19.1 million and $210.5 million, respectively, which will expire in the years 2030 through 2045. The Company also has state research and development tax credit carryforwards of $12.2 million. Deferred tax assets for these carryforwards are subject to a full valuation allowance.
The Company permanently reinvests the earnings of its foreign subsidiaries except for its U.K. operations. The determination of the amount of the unrecognized of the deferred tax liability related to the undistributed earnings is not practicable because of the complexities associated with its hypothetical calculation.
14. Collaborative Agreements
GlaxoSmithKline
In July 2012, as amended in November 2013, the Company entered into an agreement with GlaxoSmithKline ("GSK"), pursuant to which Amicus obtained global rights to develop and commercialize Galafold® as a monotherapy and in combination with ERT for Fabry disease (“Collaboration Agreement”). Under the terms of the Collaboration Agreement, GSK is eligible to receive post-approval and sales-based milestones up to $40 million, as well as tiered royalties in the mid-teens in eight major markets outside the U.S.
As of December 31, 2025, the milestone payments due to GSK were $1.5 million and are recorded within accrued expenses and other current liabilities in the Consolidated Balance Sheets. Sales based tiered royalties due to GSK are recorded within the cost of goods sold in the Consolidated Statements of Operations.
For the year ended December 31, 2025, under the GSK collaboration agreement, the Company incurred approximately $35.2 million of royalty expenses, of which $10.7 million is recorded within accrued expenses and other current liabilities in the Consolidated Balance Sheets.
Dimerix Limited
On April 30, 2025, the Company entered into an exclusive license agreement with Dimerix for the commercialization of Dimerix' Phase 3 drug candidate, DMX-200, in the U.S. for treatment of FSGS and other indications. In exchange for these rights, the Company paid Dimerix an upfront payment of $30 million, which was recorded as a component of research and
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development expense in the Company's Consolidated Statement of Operations. The Company will be obligated to pay Dimerix for certain success-based development and regulatory milestones for FSGS of up to a maximum aggregate amount of $75 million, regulatory milestones for other indications of up to a maximum aggregate amount of $40 million, commercial milestones of up to a maximum aggregate amount of $445 million, and tiered royalties of DMX-200 net sales in the U.S. ranging from the low-teens to low-twenties.
Dimerix will continue to fund and execute the Phase 3 study of DMX-200 ("ACTION3"), and Amicus will be responsible for submission and maintenance of the regulatory dossier in the United States, as well as all costs of commercialization activities. Additionally, Amicus will have the exclusive rights to develop DMX-200 in other future indications in the United States. Amicus and Dimerix have formed a Joint Steering Committee to align the development and commercialization of DMX-200 in FSGS in U.S. The agreement otherwise contains terms common for an arrangement of this kind.
In December of 2025, Dimerix announced completion of enrollment for the ACTION3 (n=286).
15. Merger Agreement
On December 19, 2025, the Company entered into the Merger Agreement with BioMarin and its wholly owned acquisition subsidiary. Under the terms of the Merger Agreement and in accordance with the DGCL, if the Merger is completed, BioMarin's wholly owned acquisition subsidiary will merge with and into Amicus, and Amicus will continue as the surviving corporation as a wholly owned subsidiary of BioMarin. In addition, all shares of the Company's common stock outstanding immediately prior to closing (other than Excluded Shares or Dissenting Shares (as defined in the Merger Agreement)) will be cancelled and converted into the right to receive $14.50 per share in cash, without interest and subject to any applicable withholding of taxes. Following completion of the Merger, shares of the Company's common stock will no longer be publicly listed.
The Boards of Directors of both companies have approved the Merger Agreement. The Merger Agreement includes restrictions on the conduct of the Company's business prior to the completion of the Merger or termination of the Merger Agreement, generally requiring the Company to conduct its business in the ordinary course in all material respects. Without limiting the generality of the foregoing, unless the Company obtains BioMarin's prior written consent, and except (i) as required or expressly contemplated by the Merger Agreement, (ii) as required by applicable law, or (iii) as set forth in the confidential disclosure schedule delivered by Amicus to BioMarin in connection with the execution of the Merger Agreement, the Company may not, among other things, incur additional indebtedness, issue additional shares of the Company's common stock outside of existing equity plans, repurchase shares, declare or pay dividends, acquire or dispose of assets or businesses (subject to limited exceptions), enter into or amend certain contracts and make capital expenditures above specified thresholds.
At the effective time of the Merger, the Company's outstanding equity awards will be treated as follows: (i) each option to purchase shares of the Company's common stock (a "Company Option") that is outstanding and unexercised (whether or not vested) and has a per share exercise price that is less than the Merger Consideration (each, an "In the Money Option") will be cancelled and converted into the right to receive a cash payment (without interest and less applicable tax withholdings and other authorized deductions) equal to the product of (a) the excess of (x) the Merger Consideration over (y) the exercise price payable per share under such In the Money Option, multiplied by (b) the total number of shares of the Company's common stock subject to such In the Money Option immediately prior to the effective time of the Merger (the "Effective Time") (without regard to vesting); (ii) each Company Option other than an In the Money Option that is outstanding and unexercised as of the Effective Time, whether or not vested, will be cancelled with no consideration payable in respect thereof; (iii) each then-outstanding time-vesting restricted stock unit with respect to shares of the Company's common stock granted under its equity plans (each, a "Company RSU") will be cancelled and converted into the right of the holder thereof to receive a cash payment (without interest and less applicable tax withholdings and other authorized deductions) equal to the product of (a) the Merger Consideration multiplied by (b) the number of shares of the Company's common stock subject to such Company RSU; and (iv) each then-outstanding performance-vesting restricted stock unit with respect to shares of the Company's common stock granted under its equity plans (each, a "Company PSU"), whether vested or unvested, will be cancelled and converted into the right to receive a cash payment (without interest and less applicable tax withholdings and other authorized deductions) equal to the product of (rounded down to the nearest whole number) (i) the number of shares of the Company's common stock subject to such Company PSU immediately prior to the Effective Time at specified levels of performance, without any pro-ration, as of immediately prior to the Effective Time multiplied by (ii) the Merger Consideration.
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The completion of the pending transaction with BioMarin is conditioned upon (i) the adoption of the Merger Agreement by the holders of at least a majority of the outstanding shares of the Company's common stock, (ii) the receipt of certain regulatory approvals, including expiration or termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 and the receipt of any clearance, consent or affirmative approval applicable to the transaction under antitrust laws and foreign direct investment laws of any non-U.S. or supranational governmental bodies listed in the confidential disclosure schedule delivered by Amicus to BioMarin in connection with the execution of the Merger Agreement, including for certain European countries and the Japanese competition authority, (iii) the absence of certain legal restraints preventing or otherwise making illegal the consummation of the Merger, (iv) the accuracy of certain representations and warranties that we made and compliance by us with certain covenants contained in the Merger Agreement, subject to qualifications, (v) there not having been a "Company Material Adverse Effect" (as defined in the Merger Agreement) with respect to us since the date of the Merger Agreement, and (vi) other customary conditions.
The Merger Agreement may be terminated under certain specified circumstances, including, but not limited to, a change in the recommendation of the Company's Board of Directors or a termination of the Merger Agreement by us to enter into an agreement for a "Superior Offer," as defined in the Merger Agreement. We could also be required to pay BioMarin a termination fee of $175 million if the Merger Agreement is terminated under specific circumstances described in the Merger Agreement, including in connection with Amicus accepting a Superior Offer or due to the Company's Board of Directors changing or withdrawing its recommendation in favor of the Merger.
16. Legal Proceedings
In the fourth quarter of 2022, the Company received Paragraph IV Certification Notice Letters from Teva Pharmaceuticals USA, Inc. ("Teva"), Aurobindo Pharma Limited ("Aurobindo"), and Lupin Limited ("Lupin") in connection with Abbreviated New Drug Applications (“ANDA”) filed with the FDA requesting approval to market generic Galafold®. In November 2022, the Company filed four lawsuits against Teva, Lupin, and Aurobindo in the U.S. District Court for the District of Delaware (the "District Court") for infringement of its Orange Book-listed patents. In the fourth quarter of 2023, a stipulation order to stay litigation with respect to Lupin was ordered. Additionally, in the first quarter of 2024, a stipulation was filed with the court and approved by the presiding judge, whereby the parties agreed to accept the Company’s definition of the terms that were in dispute. As such, the scheduled Markman hearing was deemed unneeded and cancelled.
In October 2024, the Company entered into a non-exclusive, non-transferable, royalty-free, fully paid-up license with Teva which will allow Teva to market its generic version of Galafold® in the United States beginning on January 30, 2037, or earlier in certain circumstances. In accordance with the license agreement, a consent judgment and permanent injunction was entered with the District Court and all Hatch-Waxman litigation between Amicus and Teva has been terminated. As required by law, Amicus and Teva have submitted the confidential license agreement to the U.S. Federal Trade Commission and the U.S. Department of Justice for review.
From September 29, 2025 to October 1, 2025, the District Court held a three-day trial to hear evidence and argument as to the disputed patent issues between Aurobindo and Amicus.
On December 19, 2025, Amicus announced that it had resolved the patent litigation with Aurobindo and Lupin. In connection with the resolution of the patent litigation, Amicus entered into non-exclusive, non-transferable, royalty-free licenses with Aurobindo and Lupin respectively, which will allow Aurobindo and Lupin to market their respective generic versions of Galafold® in the United States beginning on January 30, 2037, or earlier in certain circumstances. As required by law, the companies have submitted the confidential license agreements to the U.S. Federal Trade Commission and the U.S. Department of Justice for review. In accordance with the license agreements, the parties have terminated all ongoing Hatch-Waxman litigation between Amicus and Aurobindo and Lupin regarding Galafold® patents pending in the District Court.
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17. Basic and Diluted Net Loss per Common Share
The following table provides a reconciliation of the numerator and denominator used in computing basic and diluted net loss attributable to common stockholders per common share:
| Years ended December 31, | ||||||||||||||||||||
(in thousands, except per share amounts) | 2025 | 2024 | 2023 | |||||||||||||||||
| Numerator: | ||||||||||||||||||||
| Net loss attributable to common stockholders | $ | ( | $ | ( | $ | ( | ||||||||||||||
| Denominator: | ||||||||||||||||||||
| Weighted average common shares outstanding — basic and diluted | ||||||||||||||||||||
Dilutive common stock equivalents would include the dilutive effect of outstanding common stock options and unvested RSUs. Potentially dilutive common stock equivalents were excluded from the diluted earnings per share denominator for all periods because of their anti-dilutive effect. Weighted average common shares outstanding includes outstanding pre-funded warrants with an exercise price of $0.01 .
The table below presents potential shares of common stock that were excluded from the computation as they were anti-dilutive using the treasury stock method:
| Years ended December 31, | ||||||||||||||||||||
| (in thousands) | 2025 | 2024 | 2023 | |||||||||||||||||
| Options to purchase common stock | ||||||||||||||||||||
| Unvested restricted stock units | ||||||||||||||||||||
| Total number of potentially issuable shares | ||||||||||||||||||||
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18. Subsequent Events
As previously disclosed in the Merger Proxy Statement, on January 21, 2026, the Company and BioMarin each filed a Premerger Notification and Report Form under the HSR Act with the Antitrust Division of the U.S. Department of Justice and the U.S Federal Trade Commission (the "FTC") in connection with the Merger. On February 11, 2026, the FTC granted early termination of the waiting period under the HSR Act.
The termination of the waiting period under the HSR Act satisfies one of the conditions to the completion of the pending transaction with BioMarin. Completion of the pending transaction with BioMarin remains subject to the satisfaction or waiver of other customary closing conditions, including the adoption of the Merger Agreement by the holders of at least a majority of the outstanding shares of the Company’s common stock and the receipt of any clearance, consent or affirmative approval applicable to the transaction under antitrust laws and foreign direct investment laws of any non-U.S. or supranational governmental bodies listed in the confidential disclosure schedule delivered by the Company to BioMarin in connection with the execution of the Merger Agreement, including for certain European countries and the Japanese competition authority. The transaction is expected to close in the second quarter of 2026, subject to regulatory clearances, approval by the Company’s stockholders and other customary closing conditions.
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Item 9. CHANGES IN AND DISAGREEMENTS WITH ACCOUNTANTS ON ACCOUNTING AND FINANCIAL DISCLOSURE.
None.
Item 9A. CONTROLS AND PROCEDURES.
Evaluation of Disclosure Controls and Procedures
Our management, with the participation of our principal executive officer and principal financial officer, evaluated the effectiveness of our disclosure controls and procedures as of December 31, 2025. The term "disclosure controls and procedures," as defined in Rules 13a-15(e) and 15d-15(e) under the Exchange Act, means controls and other procedures of a company that are designed to ensure that information required to be disclosed by us in the reports that we file or submit under the Exchange Act is recorded, processed, summarized and reported within the time periods specified in the SEC rules and forms. Disclosure controls and procedures include, without limitation, controls and procedures designed to ensure that information required to be disclosed by a company in the reports that it files or submits under the Exchange Act is accumulated and communicated to the Company's management, including its principal executive and principal financial officers, as appropriate to allow timely decisions regarding required disclosure. Management recognizes that any controls and procedures, no matter how well designed and operated, can provide only reasonable assurance of achieving their objectives and management necessarily applies its judgment in evaluating the cost-benefit relationship of possible controls and procedures. Based on the evaluation of our disclosure controls and procedures as of December 31, 2025, our principal executive officer and principal financial officer concluded that, as of such date, our disclosure controls and procedures were effective at the reasonable assurance level.
There have been no changes in our internal controls over financial reporting during the fourth quarter of the year ended December 31, 2025 that have materially affected, or are reasonably likely to materially affect, our internal controls over financial reporting.
Management's Report on Internal Control Over Financial Reporting
The information required by this section which includes the "Management's Report on Consolidated Financial Statements and Internal Control over Financial Reporting" and the "Report of Independent Registered Public Accounting Firm" are incorporated by reference from "Item 8. Financial Statements and Supplementary Data."
Item 9B. OTHER INFORMATION.
Rule 10b5-1 Trading Plans
For the quarterly period covered by this report, no director or officer (as defined in Rule 16a-1(f) under the Exchange Act) has adopted terminated
Item 9C. DISCLOSURE REGARDING FOREIGN JURISDICTIONS THAT PREVENT INSPECTIONS.
Not applicable.
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PART III
Item 10. DIRECTORS, EXECUTIVE OFFICERS OF THE REGISTRANT AND CORPORATE GOVERNANCE.
Our Board of Directors
Our Certificate of Incorporation and By-laws provide that our business is to be managed by or under the direction of our Board. Our Board is divided into three classes and one class is elected at each Annual Meeting of Stockholders to serve for a three-year term. Our Board consists of nine members and is divided amongst the three classes as follows:
●The Class I directors are Mr. Campbell and Ms. Bleil, and their term would expire at the 2026 Annual Meeting of Stockholders;
●The Class II directors are Messrs. Wheeler and Whitman and Dr. Roberts, and their term would expire at the 2027 Annual Meeting of Stockholders; and
●The Class III directors are Messrs. Kelly, Raab and Sblendorio and Ms. McGlynn, and their term would expire at the 2028 Annual Meeting of Stockholders.
Our Certificate of Incorporation and By-laws provide that the authorized number of directors may be changed only by resolution of the Board. Our Board has authorized that the maximum size of the Board be set at twelve members.
Name | Age | Position | ||||||
Lynn D. Bleil (1)(2) | 62 | Director | ||||||
Bradley L. Campbell | 50 | Director | ||||||
Michael A. Kelly (3)(4) | 69 | Director | ||||||
Margaret G. McGlynn (5)(6) | 66 | Director | ||||||
Michael G. Raab (6)(7) | 61 | Director | ||||||
Eiry W. Roberts, M.D. (2)(4) | 62 | Director | ||||||
Glenn P. Sblendorio (2)(8) | 70 | Director | ||||||
Craig A. Wheeler (3)(9) | 65 | Director | ||||||
Burke W. Whitman (3)(6) | 69 | Director | ||||||
(1)Chair of the Nominating and Corporate Governance Committee
(2)Member of the Compensation and Leadership Development Committee
(3)Member of Audit and Compliance Committee
(4)Member of the Science and Technology Committee
(5)Chair of the Compensation and Leadership Development Committee
(6)Member of Nominating and Corporate Governance Committee
(7)Chairman of the Board
(8)Chair of the Audit and Compliance Committee
(9)Chair of the Science and Technology Committee
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 | Lynn D. Bleil has served as a member of the Board since September 2018. Ms. Bleil led the West Coast Healthcare Practice of McKinsey & Company, a multinational strategy and management consulting firm, and was a core leader of McKinsey’s worldwide Healthcare Practice before her retirement as a Senior Partner in 2013, after 25 years at the firm. Currently, Ms. Bleil serves as a member of the Board of Directors of Sonova Holding AG (VX: SOON) and Alcon AG (NYSE: ALC). Her prior directorships include DST Systems, Inc. (NYSE: DST), Auspex Pharmaceuticals (Nasdaq: ASPX), and Stericycle, Inc. (Nasdaq: SRCL). Ms. Bleil is also the Chair of the Intermountain Wasatch Back Hospitals Community Board, a non-profit organization. Ms. Bleil received her B.S.E. in Chemical Engineering from Princeton University and her M.B.A. from the Stanford Graduate School of Business. | ||||
Skills and Qualifications: Ms. Bleil is an experienced Director who brings more than three decades of experience in the broader healthcare and biopharma industries, having advised numerous executives and Boards in the sector on strategic, organizational, and operational issues. She has broad expertise in healthcare strategy, business development, go-to-market strategies, reimbursement, and policy.
 | Bradley L. Campbell is the Company’s President and Chief Executive Officer and has served as a member of the Board since June 2018. From January 2015 until his promotion to Chief Executive Officer in August 2022, Mr. Campbell served as President and Chief Operating Officer. He brings over 20 years of experience in the Orphan Drug industry. Mr. Campbell joined Amicus in 2006 and, prior to becoming CEO led the global organization responsible for the commercialization of Galafold®. He also oversaw the Technical Operations, Market Access, Program Management, Clinical Operations, and Regulatory Affairs functions. Mr. Campbell currently serves on a number of Boards including the Corporate Advisory Board for the National Tay-Sachs and Allied Diseases Association, the board of the Biotechnology Innovation Organization and the Duke-Margolis Advisory Board. He previously served on the Boards of Gennao Bio and ARYA Sciences Acquisition Corp III, a healthcare focused Special Purpose Acquisition Vehicle, as well as Progenics Pharmaceuticals (Nasdaq: PGNX) from 2016 until its successful acquisition by Lantheus Holdings in 2020. Prior to joining Amicus, Mr. Campbell spent time in various commercial and business development roles at Genzyme and Bristol-Myers Squibb and as a strategy consultant for Marakon Associates. He received a B.A. in Public Policy Studies from Duke University and an M.B.A. from Harvard Business School. | ||||
Skills and Qualifications: Mr. Campbell has significant experience within the pharmaceutical industry, much of which has been focused on rare diseases, including expertise in corporate development, strategic planning, business operations, sales, and marketing. His experience, as well as his prior service on the Board of Directors of other publicly held companies in the pharmaceutical industry, provide valuable contributions to the Company as we continue our ongoing expansion as a fully integrated global commercial company. He also provides our Board with in-depth knowledge of our company through the day-to-day leadership of our executives, all of which enables him to make meaningful contributions to the Board.
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 | Michael A. Kelly has served as a member of the Board since December 2020. Mr. Kelly is a former senior executive of Amgen, Inc. (Nasdaq: AMGN), a multinational biopharmaceutical company, and is currently acting as Founder & President of Sentry Hill Partners, LLC, a global life sciences transformation and management consulting business founded by Mr. Kelly in 2018. Mr. Kelly has more than two decades of executive experience as a senior leader in the life sciences industry serving in various strategic finance and operations positions at Amgen Inc., most recently as Senior Vice President, Global Business Services and Vice President & CFO, International Commercial Operations. Mr. Kelly has also held positions at Biogen, Inc. (Nasdaq: BIIB), Tanox, Inc., and Monsanto Life Sciences, a division of the Nutrasweet Kelco Company. Mr. Kelly currently serves as an independent member of the Board of Directors of Prime Medicine, Inc. (Nasdaq: PRME), DMC Global, Inc. (Nasdaq: BOOM), NeoGenomics, Inc. (Nasdaq: NEO), and NChroma Bio, a private company. Mr. Kelly previously served on the Boards of Directors for Aprea Therapeutics, Inc. (Nasdaq: APRE) and Hookipa Pharma, Inc. (Nasdaq: HOOK). He also serves on the Council of Advisors and was the former audit committee chairman for Direct Relief, a humanitarian aid organization focused on health outcomes and disaster relief. Mr. Kelly holds a BSc in business administration from Florida A&M University, concentrating in Finance and Industrial Relations. | ||||
Skills and Qualifications: Mr. Kelly brings more than two decades of leadership experience in the life sciences industry and a wealth of knowledge and background in managing, financing, and growing global healthcare and biotechnology companies to the Board. He has served in various strategic finance and commercial operations positions, including, Founder and President of a global life sciences transformation and management consulting business, Chief Financial Officer, and Board member of multiple biotechnology companies. Mr. Kelly also has extensive experience in developing and executing global corporate strategies for multi-product biotechnology organizations, a special skill set in organizational development, as well as leadership experience in humanitarian aid facing organizations focused on health outcomes and disaster relief.
 | Margaret G. McGlynn has served as a member of the Board since October 2009. She retired from Merck & Company, Inc. (NYSE: MRK) (“Merck”), a multinational pharmaceutical company, after 26 years and serving in roles including President of Global Vaccines and Infectious Disease and President, U.S. Hospital and Specialty Products. She also served in a variety of executive leadership roles in global and U.S. marketing, sales, and managed care. Following her retirement from Merck, Ms. McGlynn served as Chief Executive Officer and President of The International AIDS Vaccine Initiative. Currently, Ms. McGlynn serves as Chair of the Board of Directors of Novavax, Inc. (Nasdaq: NVAX). Previously, she served on the Boards of Vertex Pharmaceuticals Inc. (Nasdaq: VRTX), Air Products and Chemicals, Inc. (NYSE: APD) and Orphan Technologies Ltd. She is also Chair Emeritus of the Board of HCU Network America, a non-profit which provides advocacy and supports research for patients affected by the rare disease homocystinuria, and is a Trustee of University at Buffalo Foundation. Ms. McGlynn holds a B.S. in Pharmacy and a M.B.A. in Marketing and an honorary doctorate in sciences from the State University of New York at Buffalo. | ||||
Skills and Qualifications: Ms. McGlynn has significant leadership experience in the pharmaceutical industry, including her service as a senior executive of Merck where she led commercialization across several therapeutic areas and geographies and managed large organizations. This experience, combined with her service on biopharmaceutical company boards and a rare disease patient advocacy organization, gives her important insights into Amicus’s business and a comprehensive understanding of compensation management and the relationship of compensation practices to the organization and its development.
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 | Michael G. Raab has served as a member of the Board of Directors since May 2004, as Lead Independent Director September 2018-March 2024, and as Chair of the Board since March 2024. Mr. Raab has served as President and Chief Executive Officer of Ardelyx, Inc. (Nasdaq: ARDX), a biopharmaceutical company since March 2009. Mr. Raab previously served as a partner of New Enterprise Associates (‘‘NEA’’) from June 2002 until December 2008, with a focus on healthcare investing. From 1999 to 2002, he was Senior Vice President, Therapeutics and General Manager, Renagel® at Genzyme Corporation. Mr. Raab currently serves as a member of the Board of Directors of Ardelyx, Inc. and Chairman of Tempest Therapeutics, Inc. (Nasdaq: TPST), a San Francisco based clinical stage biotechnology company advancing small molecule therapeutics that modulate anti-tumor pathways. He also serves on the Emerging Companies and Health Section Governing Boards of the Biotechnology Innovation Organization. Mr. Raab holds a B.A. from DePauw University. | ||||
Skills and Qualifications: Mr. Raab has significant experience in drug development and commercialization of products in the rare diseases, cardio renal, and GI diseases. He also has extensive management experience in the biopharmaceutical industry serving as Chief Executive Officer of a late-stage biopharmaceutical company and from his prior time overseeing NEA investments in pharmaceuticals and biotechnology. Mr. Raab also brings a global perspective and an integrity-based approach to governance matters and devotes substantial time and attention to all Amicus matters.
 | Eiry W. Roberts, M.D., has served as a member of our Board since June 2021. Dr. Roberts currently serves as a strategic advisor to Neurocrine Biosciences, Inc. (Nasdaq: NBIX), where she previously served as Chief Medical Officer, and is a former senior executive of Eli Lilly and Company (NYSE: LLY), a multinational pharmaceutical company. She has over 30 years of pharmaceutical drug development experience, ranging across all phases of development from research through commercialization, spanning multiple therapeutic areas. Prior to Neurocrine, Roberts spent 26 years at Eli Lilly, during which she advanced through various senior and executive level roles, concluding her tenure as Vice President in Research & Development. Roberts served as the Chair of the Medical Review Committee at Eli Lilly. She is a member of the Healthcare Business Women’s Association and an Adjunct Professor of Medicine at Indiana University, Department of Clinical Pharmacology. She previously served on the Springboard Ventures Steering Committee and was a member of the Indiana Health Forum. She has non-profit Board experience, previously serving on the Board of the Indianapolis Children’s Choir and the St. Richard’s Episcopal School Board of Trustees. Dr. Roberts is an M.D. trained in pharmacology and medicine in the United Kingdom, qualifying from the University of London. Dr. Roberts continued her post-graduate clinical training in clinical pharmacology and cardiology at St. Bartholomew’s Hospital and at the Royal London Hospital. | ||||
Skills and Qualifications: Dr. Roberts brings more than 30 years of healthcare industry experience to the Board, spanning the areas of pharmaceutical drug development, regulatory affairs, pricing, and access. She has immense experience in leading therapeutic programs through all phases of the drug development process, regulatory frameworks, and product commercialization. She has an extensive background in medicine and experience as a Chief Medical Officer of a biopharmaceutical company. The culmination of her skills and experience adds important insight into the Amicus business and its development into a leading global commercial organization.
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 | Glenn P. Sblendorio has served as a member of the Board since June 2006. Mr. Sblendorio most recently was Chief Executive Officer and member of the Board of Directors of IVERIC bio, Inc. (Nasdaq: ISEE), formerly Ophthotech Corporation (Nasdaq: OPHT), a biopharmaceutical company, from July 2017 to its acquisition by Astellas Pharma in 2023. Prior to IVERIC, Mr. Sblendorio was President and Chief Financial Officer of The Medicines Company (Nasdaq: MDCO) from March 2006 through March 2016 and was a member of the Board of Directors of the Medicines Company from July 2011 through December 31, 2015. Before joining The Medicines Company, Mr. Sblendorio was Executive Vice President and Chief Financial Officer of Eyetech Pharmaceuticals, Inc. from February 2002 until it was acquired by OSI Pharmaceuticals, Inc. in November 2005. In addition, from 1998 through 2000, Mr. Sblendorio served as a Managing Director of MPM Capital Advisors. Mr. Sblendorio currently serves as Chair of the Board of Directors of Mineralys Therapeutics (Nasdaq: MLYS), is a member of the Board of Directors of 4D Molecular Therapeutics, Inc. (Nasdaq: FDMT), serves as Chairman of the Board of Nanoscope Therapeutics, a private company, and is a member of the Board of Directors of RetinaNova, also a private company. Previously, he served as a member of the board of Directors of Intercept Pharmaceuticals, Inc. (Nasdaq: ICPT) until it was acquired in November 2023. Mr. Sblendorio received his B.B.A. from Pace University, his M.B.A. from Fairleigh Dickinson University and is a graduate of the Harvard Advanced Management Program. | ||||
Skills and Qualifications: Mr. Sblendorio has significant corporate leadership experience, industry knowledge and demonstrated knowledge of financial and financing matters through his prior experience in leading pharmaceutical companies. He brings substantial expertise in the management of financial and compliance risks associated with global pharmaceutical operations and financial management strategies. Mr. Sblendorio’s specific expertise includes his service on other boards, and he devotes significant time to Amicus matters both in scheduled meetings and with management and the auditors. He is the “audit committee financial expert” as defined in the SEC regulations, with particular expertise in the matters faced by the audit committee of a company with its commercial revenue guidance, geographic expansion and related expenses.
 | Craig A. Wheeler has served as a member of the Board since June 2016. He is the CEO of Headwaters Biotech Advisors LLC, where he serves as an advisor to executives in the Biotech industry. Mr. Wheeler recently completed a 14-year tenure as President and Chief Executive Officer of Momenta Pharmaceuticals (Nasdaq: MNTA), where he grew the company from a startup, through multiple product launches, and ultimately to a $6.5 billion acquisition by Johnson and Johnson in the fall of 2020. In 2011, he was an EY Entrepreneur of the Year Regional Award winner. In May 2012, the Boston Globe named Momenta the number one company in their annual Globe 100 survey of top performing companies. Prior to joining Momenta, Mr. Wheeler was President of Chiron Biopharmaceuticals where, during his five-year tenure, he ran a fully integrated 2,500-person global pharma business with a global commercial organization, multiple manufacturing sites, a research organization, and a product development pipeline, more than doubling the pharmaceutical division’s global sales. Before that, he was a senior member of The Boston Consulting Group’s health care practice and worked extensively in the health care sector with focus on pharma and biotech, particularly in regard to corporate and R&D strategy. He began his career at Merck & Company, Inc.’s (NYSE: MRK) MSDRL research unit. He is currently a member of the board of Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) and has previously served as the Chairman of the Board of Avanir Pharmaceuticals, Inc. where he helped oversee the transition of the company from a research-based platform to a fully integrated CNS pharmaceutical company until 2015 when it was acquired by Otsuka Pharmaceuticals for $3.5 billion. Mr. Wheeler received his B.S. and M.S. in chemical engineering from Cornell University and his M.B.A. from the Wharton School of the University of Pennsylvania. | ||||
Skills and Qualifications: Mr. Wheeler has extensive pharmaceutical industry knowledge and leadership experience, including his demonstrated expertise in drug development, manufacturing, and the technical issues facing growing biopharmaceutical companies. This background enables him to make significant contributions as the head of the Science and Technology
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Committee, while his overall life science experience and leadership enables him to contribute to the Audit and Compliance Committee, as well as the Board as a whole.
 | Burke W. Whitman has served as a member of the Board since June 2019. He also serves as the Chief Executive of Colmar Holdings (a private holding company); a member of the Boards of Directors of Omega Healthcare Investors (NYSE: OHI) (Chair of the Compensation Committee), the Marine Corps Heritage Foundation (Vice Chair of the Board), and the Buckhead Coalition (Executive Committee); a board member of the Piedmont Health Foundation; and a national member of Business Executives for National Security. Previously Mr. Whitman served as both a corporate Chief Executive Officer and a U.S. Marine Corps General Officer. In military service for 34 years (1985 to 2019, nearly half on active duty, most recently 2009 to 2018), he commanded units at every level, led multiple combat deployments, served as Commanding General of a Marine Division and of Marine Forces and as a General Officer with the U.S. Secretary of Defense, and retired as a Major General and the service’s senior reserve officer. Concurrently, in business for 21 years (1988-2009), he served as Chief Executive Officer (initially Chief Operating Officer) of Health Management Associates (NYSE: HMA), Chief Financial Officer of Triad Hospitals (NYSE: TRI), President of Deerfield Healthcare (private), Vice President of Almost Family (Nasdaq: AFAM), and an Investment Banker with Morgan Stanley (NYSE: MS). Institutional Investor Magazine named him a repeat Best CFO and Best CEO; the nation awarded him two dozen military and combat decorations including the Distinguished Service Medal. In volunteer service, Mr. Whitman has served on the Boards of the Federation of American Hospitals, Toys for Tots Foundation, Reserve Forces Policy Board, Marine Corps University, and Lovett School. He holds a BA from Dartmouth College, an MBA from Harvard Business School, a Master in Strategic Studies degree from the United States Army War College, and a Master in Ministry degree from Nashotah Theological Seminary. | ||||
Skills and Qualifications: Mr. Whitman is an experienced executive and board leader of national and global organizations in health, defense, education, finance, and real estate. His broad knowledge of the domestic and international healthcare sector, specific experience in strategic finance and growth, and skill in organizational leadership and governance, provide a multifaceted perspective to our global biopharma business. These qualifications have made him an integral member of the board and both the Audit and Compliance and Nominating and Corporate Governance Committees.
Committee Memberships
Directors | Independent | Age | Director Since | Audit and Compliance | Compensation and Leadership Development | Nominating and Corporate Governance | Science and Technology | ||||||||||||||||
Lynn D. Bleil | ü | 62 | 2018 | ● | C | ||||||||||||||||||
Bradley L. Campbell | 50 | 2018 | |||||||||||||||||||||
Michael A. Kelly | ü | 69 | 2020 | ● | ● | ||||||||||||||||||
Margaret G. McGlynn | ü | 66 | 2009 | C | ● | ||||||||||||||||||
Michael G. Raab (CH) | ü | 61 | 2004 | ● | |||||||||||||||||||
Eiry W. Roberts, M.D. | ü | 62 | 2021 | ● | ● | ||||||||||||||||||
Glenn P. Sblendorio | ü | 70 | 2006 | C | ● | ||||||||||||||||||
Craig A. Wheeler | ü | 65 | 2016 | ● | C | ||||||||||||||||||
Burke W. Whitman | ü | 69 | 2019 | ● | ● | ||||||||||||||||||
“CH” Chairman of the Board
“C” Committee Chair
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Executive Officers
The following is a brief summary of the background of each of our executive officers, except for Mr. Campbell whose background may be found above under “Our Board of Directors”:
Simon Harford, 65, joined Amicus in August 2023 and currently serves as Chief Financial Officer. He brings extensive finance experience in the pharmaceutical and healthcare industry both in the U.S. and internationally. Prior to joining Amicus, Mr. Harford served as Chief Financial Officer of Boston-based biotech Albireo Pharma Inc., a rare pediatric liver disease company, from October 2018 until its sale to Ipsen S.A. in March 2023. Previously he was Chief Financial Officer at PAREXEL International Corporation, a leading global clinical research organization, where he led the financial aspects of the transition from a public to private-equity owned company. Mr. Harford has spent most of his career in the pharmaceutical industry including eight years at GlaxoSmithKline plc based at their headquarters in London culminating in his role as SVP Finance, Global Pharmaceuticals with responsibility for the finance function of the global pharmaceutical business. Earlier in his career, he spent 20 years at Eli Lilly and Company in numerous senior leadership roles in the U.S. and Europe including Head of Investor Relations, European CFO and Corporate Controller. Mr. Harford has an MBA from the University of Virginia’s Darden School of Business.
Ellen S. Rosenberg, 63, has served as our Chief Legal Officer and Corporate Secretary since December 2018 and our General Counsel and Corporate Secretary since February 2016. Prior to joining Amicus, she served as a Senior Vice President of Shire Pharmaceuticals. Prior to Shire, Ms. Rosenberg was Associate General Counsel for the Metabolic Endocrinology division at EMD Serono Inc., the U.S. affiliate of Merck KGaA. Ms. Rosenberg brings extensive and broad ranging legal experience in the biopharmaceutical and medical device industry including mergers and acquisitions, licensing, corporate governance, product launches, risk management, litigation, investigations, intellectual property, and compliance matters. Ms. Rosenberg also has significant experience building and developing legal teams and the in-house legal function. Ms. Rosenberg received a B.A. from the University of Connecticut and a J.D. from the University of Pennsylvania Carey Law School.
David M. Clark, 51, has served as our Chief People Officer since October 2018. Mr. Clark was previously Vice President of Global Human Resources (HR) at Alibaba Group, headquartered in Hangzhou, China, from September 2016 to August 2018. Prior to that, Mr. Clark spent eight years at American Express, where he was Senior Vice President of Human Resources and Chief Learning Officer. While there, Mr. Clark was a senior HR Business Partner and led the transformation of learning, leadership development and performance management. Previously, Mr. Clark was a Commissioned Officer on the White House senior staff. As Deputy Assistant to the President of the United States, he led the recruitment and development of the 4,000 most senior leaders in the U.S. government. Mr. Clark received a B.S. in political science from Indiana State University. He is an Eagle Scout and served on the National Executive Board of the Boy Scouts of America. Mr. Clark is also a Chairman-Emeritus of the Board of the Make-A-Wish Foundation of America.
Jeffrey P. Castelli, Ph.D., 54, has served as Chief Development Officer since May 2020. Previously he served as the Company’s Chief Portfolio Officer and Head of Gene Therapy and has been employed with Amicus since July 2005. Dr. Castelli has over 20 years of experience in the Biotech and Orphan Drug industry, focused on rare disease research and development of medicines from discovery through market authorization. In his current capacity, he provides strategic leadership across all R&D activities, including direct oversight of Regulatory, Science, Clinical Research, Clinical Operations, and Medical Affairs. While at Amicus, he has had responsibility for a number of different functions including program management, portfolio planning and the gene therapy business. Dr. Castelli previously served as a healthcare strategy consultant at Health Advances LLC and worked in business development at Neose Pharmaceuticals Inc. He received a B.S. from West Chester University and a Ph.D. from the University of Pennsylvania and is an author on numerous publications and patents in the field of rare disease drug development.
Code of Conduct
Our Code of Conduct applies to all of our employees, including our principal executive officer, principal financial, and principal accounting officer, and our non-employee directors. The text of the Code of Conduct is posted on our web site at www.amicusrx.com and will be made available to stockholders without charge, upon request, in writing to The Office of the Corporate Secretary, c/o Amicus Therapeutics, Inc. at 47 Hulfish Street, Princeton, New Jersey 08542. Disclosure regarding any amendments to, or waivers from, provisions of the Code of Conduct that apply to our directors, principal executive and
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financial and accounting officers will be included in a Current Report on Form 8-K within four business days following the date of the amendment or waiver, unless web site posting of such amendments or waivers is then permitted by the rules of Nasdaq.
Audit and Compliance Committee.
Our Board has an Audit and Compliance Committee and the current members are Messrs. Kelly, Sblendorio, Wheeler and Whitman. Mr. Sblendorio is the Chair of the Audit and Compliance Committee.
Our Board has determined that Mr. Sblendorio is a financial expert within the meaning of Item 407(d)(5) of Regulation S-K and has “accounting or related financial management expertise” within the meaning of the rules and regulations of Nasdaq. Our Audit and Compliance Committee was established in accordance with Section 3(a)(58) of the Securities Exchange Act of 1934, as amended (the “Exchange Act”). Our Audit and Compliance Committee assists our Board in its oversight of the integrity of our financial statements, our independent registered public accounting firm’s qualifications, independence and the performance of our independent registered public accounting firm and our compliance program.
Nasdaq rules require that all members of the Audit and Compliance Committee be independent directors, as defined by the rules of Nasdaq and the SEC. Our Board has determined that all the members of the Audit and Compliance Committee satisfy the independence requirements for service on the Audit and Compliance Committee.
A copy of the Audit and Compliance Committee written charter is publicly available on our web site at www.amicusrx.com.
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Item 11. EXECUTIVE COMPENSATION.
Compensation Discussion and Analysis (CD&A)
On December 19, 2025, we entered into a merger agreement with BioMarin Pharmaceutical Inc. (“BioMarin”), upon the consummation of which BioMarin will acquire the Company (this acquisition is referred to below as the “Transaction”). Below we describe the compensation actions that the Compensation and Leadership Development Committee took with respect to our named executive officers in 2025, the bulk of which occurred prior to our entry into the merger agreement. In addition, we discuss certain updates that were subsequently made in 2025 with respect to the compensation of our named executive officers in connection with the contemplated Transaction. More information on the impact of the contemplated Transaction upon the compensation of our named executive officers can be found in the Company's definitive merger proxy statement, filed with the SEC on February 2, 2026 (the "Merger Proxy Statement").
Note that the information contained in this Form 10-K may differ from the information presented in past filings or the Merger Proxy Statement due to differences in disclosure requirements, facts or assumptions herein and therein, respectively.
Executive Compensation
We describe our executive compensation program below and provide an analysis of the compensation paid and earned in 2025 by our “named executive officers” – our Chief Executive Officer, Chief Financial Officer, and our three other most highly compensated executive officers. For 2025, our named executive officers are:
●President and Chief Executive Officer, Bradley L. Campbell;
●Chief Financial Officer, Simon Harford;
●Chief Legal Officer and Corporate Secretary, Ellen S. Rosenberg;
●Chief People Officer, David M. Clark; and
●Chief Development Officer, Jeffrey P. Castelli, Ph.D.
“Say on Pay” Consideration
At our 2025 annual meeting of stockholders, approximately 97% of the shares voted at the meeting approved, on an advisory basis, the compensation of the named executive officers. Considering stockholders’ strong support of our most recent say-on-pay proposal, the Compensation and Leadership Development Committee did not undertake fundamental changes to our executive compensation programs following the 2025 annual meeting of stockholders. Nonetheless, we continued to solicit the input of our stockholders and in 2025 our investor relations team proactively engaged with major stockholders, representing approximately 60% of shares outstanding, on the Company’s pay practices. As evidenced by the voting detailed above, the vast majority of the shares voted to approve the ‘say on pay’ advisory proposal and the Compensation and Leadership Development Committee continues to focus on pay practices that align compensation with performance. The Compensation and Leadership Development Committee monitors and considers the results of the annual advisory “say on pay” proposal and feedback received from stockholders. In light of the impending Transaction and the likelihood that we will not hold an Annual Meeting of Stockholders for 2026, we do not expect to conduct an annual advisory say on pay vote in 2026.
Objectives and Philosophy of Executive Compensation
The Compensation and Leadership Development Committee, in consultation with the Board, is responsible for establishing, implementing, and overseeing our overall compensation strategy and policies, including our executive compensation program, in a manner that supports our business objectives. The Compensation and Leadership Development Committee evaluates our compensation program, taking into consideration best practices and emerging trends, stockholder input as well as data and feedback provided by our independent executive compensation consultant, Pay Governance.
We are a leading, global biotechnology company with a clear and compelling mission to develop and deliver transformative medicines for people living with rare diseases. We operate in an extremely competitive, rapidly changing and
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heavily regulated industry, and the long-term success of our business requires a high degree of innovation and adaptability. We believe that the skill, talent, and dedication of our executive officers are critical factors affecting our long-term success, especially at this critical time in our history as we execute our business strategy. Therefore, our compensation program for our executive officers, including our named executive officers, is designed to attract, retain, and motivate the best possible executive talent. Utilizing a pay-for-performance compensation philosophy, we have designed a program that provides the ability to differentiate the total compensation mix of our named executive officers based on their demonstrated performance and their potential to contribute to our long-term success.
Our compensation philosophy is to:
●provide our executives a competitive total compensation opportunity relative to the organizations with which we compete for executive talent;
●attract and retain individuals of superior ability and managerial talent who can successfully perform and succeed in our environment;
●increase the incentive to achieve key strategic and financial performance measures by linking compensation opportunities and actual compensation earned through our pay for performance compensation program to the achievement of corporate goals; and
●deliver pay in a cost-efficient manner that aligns employees’ compensation with stockholders’ long-term interests.
Our compensation program is designed to reward the accomplishment of our corporate goals in a manner consistent with the Company’s values, which stresses not only results but also how those results are attained. To meet the objectives of our compensation philosophy, we maintain a robust goal setting and performance management program.
When designing the annual cash incentive bonus program for 2025, the Compensation and Leadership Development Committee continued to believe that the corporate multiplier should be a significant factor in determining bonus payouts because it closely aligns our named executive officers’ compensation with the interests of our stockholders. The Compensation and Leadership Development Committee also continued to believe that including the individual multiplier for named executive officers, other than the Chief Executive Officer, as a component of such named executive officers’ bonus payouts, was important to incentivize our officers as we expand as a global commercial biotechnology company.
Individual goals for the named executive officers were approved by Mr. Campbell at the beginning of 2025 and were specific to such executive officer’s area of expertise and supported our corporate goals for the year. For 2025, annual cash incentive bonuses for our named executive officers, other than Mr. Campbell, were determined by the combination of both the corporate multiplier and an individual multiplier. For Messrs. Clark, Castelli and Harford, and Ms. Rosenberg, the attainment of individual goals was assessed within a range of 0 to 133% multiplier for each individual; this individual multiplier, along with the final corporate multiplier of 140%, was applied to the target bonus to determine final annual incentive bonus payouts.
Due to the Chief Executive Officer’s influence on the overall performance of Amicus, the Compensation and Leadership Development Committee believes it is appropriate and in the best interests of our stockholders to base the Chief Executive Officer’s cash bonus on the Board’s determination, with the Compensation and Leadership Development Committee’s recommendation, of the achievement of corporate objectives without regard to an individual multiplier. Accordingly, the attainment of Mr. Campbell’s annual incentive bonus payout was determined entirely based on the multiplication of the final corporate multiplier of 140% by his target bonus.
Risk Analysis of Compensation Policies and Practices
The Compensation and Leadership Development Committee is aware that compensation arrangements, if not properly designed, could encourage inappropriate or excessive risk taking. We believe that our overall compensation program encourages our named executive officers and other employees to focus on both short-term and long-term objectives and does not encourage excessive risk taking. Our stock options vest over multiple years and their value is not directly linked to the achievement of short term defined metrics. To enhance this posture, the Committee made the decision, starting in 2017, to award performance based restricted stock unit grants in addition to stock options and restricted stock units. In addition, cash incentive bonuses tied to the achievement of Company and individual goals have historically made up a small percentage of our
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executive officers’ total compensation package. The Nominating and Corporate Governance Committee implemented stock ownership guidelines, which ensure significant amounts of actual share ownership over time for our executive officers, mitigate excessive risk taking and foster an ownership mentality among our senior leaders. Further, we currently operate as a single business unit and therefore are not exposed to the risks that may be associated with operating through several segments, such as one business unit being significantly more profitable than another or having a compensation structure that is significantly different than that of other units.
Compensation Program Elements and Pay Level Determination
Each year, the Compensation and Leadership Development Committee reviews and determines base salaries, annual cash incentives and long-term incentive awards for all executive officers (with the Board approving the Chief Executive Officer’s compensation, after reviewing the Compensation and Leadership Development Committee’s recommendation).
In setting our executive compensation programs, the Compensation and Leadership Development Committee reviews market data at the 25th, 50th, and 75th percentiles and generally targets aggregate total direct compensation for the named executive officers as a group to approximately the 50th percentile of our peer group (as discussed below). Actual compensation levels for each named executive officer depend on factors such as individual performance, Company performance, skills/capabilities, overall impact/contribution, experience in position, criticality of position and internal equity. For 2025, the base salaries, annual cash incentives and long-term incentive awards determination for all named executive officers, excluding our Chief Executive Officer, were approved by our Compensation and Leadership Development Committee, which is comprised solely of independent directors. For the Chief Executive Officer, the base salary, annual cash incentives and long-term incentive awards were recommended by the Compensation and Leadership Development Committee to the Board following a review of peer group data; the Board then reviewed and approved the final compensation. The Compensation and Leadership Development Committee considered all the information presented (including external competitiveness, the individual’s performance, Company performance and internal equity) and applied its collective knowledge and discretion to determine the compensation for each named executive officer.
As part of the compensation determination process, the Chief Executive Officer presents to the Compensation and Leadership Development Committee an individual assessment of each named executive officer’s performance, excluding the Chief Executive Officer’s own performance, over the prior year, as well as the recommended compensation action for each such named executive officer. Based on corporate and individual performance, the Chief Executive Officer makes a compensation recommendation for each such named executive officer which includes actions on base salary, bonus (based on corporate and individual multipliers), and long-term incentive grant target value. Individual goals are designed to support the achievement of the yearly corporate goals. The Chief Executive Officer’s recommendations may also take into account input from the executive’s peers and direct reports, as appropriate. The recommendations of the Chief Executive Officer are afforded significant weight by the Compensation and Leadership Development Committee because of his familiarity with the day-to-day performance of his direct reports. However, the final determination of each executive officer’s pay, other than that of the Chief Executive Officer, is made by the Compensation and Leadership Development Committee.
In 2025, the Chief Executive Officer’s performance was assessed by all independent directors under the leadership of our Chairman. The Compensation and Leadership Development Committee bases its recommendation to the Board for the Chief Executive Officer’s compensation upon this assessment, and the final determination of the Chief Executive Officer’s Compensation is made by the Board.
Long-term incentive grants are based on an executive’s level within the organization, competitive data for our peer group, and in the case of our named executive officers, several other factors which are more fully described below under “Long-Term Incentive Programs”. Long-term incentive grants are designed to motivate and retain the executive team to best achieve the Company’s goals and implement our business strategy, thereby increasing stockholder value.
Role of Independent Compensation Consultant
The Compensation and Leadership Development Committee has engaged Pay Governance to assist the Compensation and Leadership Development Committee by providing ongoing executive compensation consulting. The Compensation and Leadership Development Committee has reviewed the independence of Pay Governance; because of the policies and procedures that Pay Governance and the Compensation and Leadership Development Committee have in place, the Compensation and Leadership Development Committee is confident that the advice it receives from executive compensation consultants at Pay
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Governance is objective and not influenced by Pay Governance’s or its affiliates’ relationships with the Company or its officers and has concluded that Pay Governance’s work does not raise any conflict of interest.
Peer Group
The Compensation and Leadership Development Committee, with the advice and analysis of its independent executive compensation consultant Pay Governance, established the peer group set forth below as a reference point for assessing named executive officer target compensation against market competitive data and relevant survey data as applicable. The Compensation and Leadership Development Committee, upon advice from Pay Governance, selected the companies that comprise our peer group through a robust screening process that considered publicly traded U.S. biopharmaceutical companies that were similar to Amicus in size, market capitalization and business operating model, and operate in geographic locations that generally have similar pay levels. Four companies, Blueprint Medicines Corporation (“Blueprint”), Exelixis Inc. (“Exelixis”), Insmed Incorporated (“Insmed”) and Ironwood Pharmaceuticals, Inc. (“Ironwood”), were removed from the peer group used in 2024. Blueprint was removed due to its acquisition by Sanofi, which closed in July 2025, Exelixis was removed due to its size no longer being appropriate for inclusion, Insmed was removed due to its larger market cap size, and Ironwood was removed due to its lower market cap. For 2025, the Compensation and Leadership Development Committee replaced these companies with BioCryst Pharmaceuticals, Inc., Kiniksa Pharmaceuticals International, plc, MannKind Corporation, and Supernus Pharmaceuticals, Inc. upon the recommendation of Pay Governance due to their similarity to Amicus based on the criteria set forth above. The Compensation and Leadership Development Committee generally reviews the peer group annually to ensure that it continues to reflect publicly traded companies of similar size and business model.
| Acadia Pharmaceuticals Inc. | Agios Pharmaceuticals, Inc. | Alkermes plc | ||||||
| Apellis Pharmaceuticals, Inc. | Axsome Therapeutics, Inc. | BioCryst Pharmaceuticals, Inc. | ||||||
| Catalyst Pharmaceuticals, Inc. | Kiniksa Pharmaceuticals International, plc | Halozyme Therapeutics, Inc. | ||||||
| Harmony Biosciences Holdings, Inc. | MannKind Corporation | Ionis Pharmaceuticals, Inc. | ||||||
| Supernus Pharmaceuticals, Inc. | Mirum Pharmaceuticals, Inc. | PTC Therapeutics, Inc. | ||||||
| Travere Therapeutics, Inc. | Ultragenyx Pharmaceuticals Inc. | |||||||
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Elements of Compensation
Our executive compensation consists primarily of base salary, annual cash incentive plan, and long-term incentive program, each of which plays an important role in our pay for performance philosophy and in achieving our compensation program objectives. For each element of compensation, we target an overall executive compensation program that is competitive with market data.
Base Salaries
Base salaries are paid to our named executive officers to provide a level of compensation that is both competitive with the external market and is commensurate with each named executive officer’s scope of responsibilities, past performance, experience, and skills. The base salary in effect at the end of the year for each of our named executive officers was as follows:
Name and Principal Position | Base Salary at December 31, | Base Salary Change | ||||||||||||
2025 | 2024 | |||||||||||||
Bradley L. Campbell | $825,000 | $770,000 | 7% | |||||||||||
President and Chief Executive Officer | ||||||||||||||
Simon Harford | $525,300 | $515,000 | 2% | |||||||||||
Chief Financial Officer | ||||||||||||||
Ellen S. Rosenberg | $535,600 | $515,000 | 4% | |||||||||||
Chief Legal Officer and Corporate Secretary | ||||||||||||||
David M. Clark | $497,948 | $483,445 | 3% | |||||||||||
Chief People Officer | ||||||||||||||
| Jeffrey P. Castelli | $512,595 | $492,880 | 4% | |||||||||||
Chief Development Officer | ||||||||||||||
The base salary increase for Mr. Campbell represents a planned multiyear ramp-up from his initial CEO salary to achieve a base that is closer to market and the base salary increases for each of our other named executive officers set forth above reflect merit increases for 2025.
Annual Cash Incentive Plan
We maintain an annual cash incentive program to motivate and reward the attainment of annual strategic, operational, financial, and individual goals. For all program participants, annual target cash incentive opportunities are expressed as a percentage of base salary, which we believe is consistent with market practice. The target bonus percentages of base salary were generally determined by level in the organization in accordance with market-based considerations and contractual entitlements.
The target bonus percentages for 2025 were as follows:
Position | 2025 Target Bonus % of Base Salary | ||||
Chief Executive Officer | 75% | ||||
Other Named Executive Officers | 45% | ||||
For 2025, bonuses awarded under the plan to our named executive officers, other than Mr. Campbell, were determined based on both the corporate multiplier and an individual multiplier. The corporate multiplier ranges from 50% to 160%, with the Compensation and Leadership Development Committee having final discretion to adjust the upper or lower limits as appropriate. For bonuses related to 2025 performance, the corporate multiplier was determined to be 140% for the reasons described below.
In order to determine bonus calculations under the plan, the target bonus for each eligible named executive officer, other than Mr. Campbell, was determined by first multiplying the officer’s target bonus percentage of base salary by 140% (the
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corporate multiplier) and then multiplying such result by his or her individual multiplier. Mr. Campbell’s bonus under the plan was determined solely by multiplying the 140% corporate multiplier by his target bonus percentage of 75% of base salary, which resulted in a 2025 bonus of approximately 105% of Mr. Campbell’s base salary. The table below titled “Calculation of Annual Cash Incentive Bonuses” further demonstrates the calculation of the 2025 annual bonuses paid to our named executive officers.
The Corporate Multiplier
On an annual basis, the Board works with management to set Company goals and objectives that are challenging and reflect an ambitious timetable for the execution of the Company’s strategies commensurate with our short and long-term business plan. The Company’s internal goals and objectives reflect complex assumptions based on internal analyses and projections and are intended to encourage the Company to pursue its business plan in an expedited manner. Once the Company’s goals and objectives are proposed, they are reviewed by the Compensation and Leadership Development Committee and then recommended for approval by the full Board. The Compensation and Leadership Development Committee periodically reviews the Company’s goals and, from time to time, may choose to recommend revisions to the Board.
The goals and objectives are set at the beginning of each year and the Compensation and Leadership Development Committee believes that their full attainment will be appropriately challenging due in part to internal and external factors. However, while total achievement of all goals and objectives may not be expected, the Compensation and Leadership Development Committee holds management accountable to significantly advance the Company’s business objectives throughout the year.
The Compensation and Leadership Development Committee reviews corporate performance against each of the pre-established targets and weighting to determine the extent to which such goals were attained. These objectives were established at the beginning of 2025 and were reflective of the corporate strategy at that time:
•Galafold®: Advance global commercialization of Galafold® with extraordinary patient focus and highest business integrity
•Pombiliti® + Opfolda®: Advance global commercialization of Pombiliti® + Opfolda® with extraordinary patient focus and highest business integrity
•Portfolio & Business Development: Advance product portfolio through label expansion, program expansion and manufacturing improvements; in-license/acquire new assets prioritizing late-clinical/commercial products
•Financial: Manage to Board-approved financial plan, growing non-GAAP profitability and obtaining positive unlevered free cash flow
•People and Culture: Advance the organization and patient-dedicated culture
In reaching its determination on the corporate multiplier for 2025 (which determination was made following the signing of the Merger Agreement), the Compensation and Leadership Development Committee considered the Company’s demonstrated achievement of the majority of these pre-established objectives, as well as the exceptional opportunity the Transaction provided to create compelling, premium value for shareholders while accelerating and expanding access to the Company’s products to patients in new markets across the world. The Compensation and Leadership Development Committee concluded that the Company had demonstrated high quality execution across the business and created meaningful value for all of its stakeholders. As such, the Compensation and Leadership Development Committee conducted a holistic review of these achievements and recommended a 140% corporate multiplier for 2025, which was approved by the Board.
The Individual Multiplier
Design
While we believe that the corporate multiplier should remain a significant factor in the bonus calculation, the Compensation and Leadership Development Committee also believes it is important to recognize and separately incentivize the individual performance of our named executive officers (excluding the role of the Chief Executive Officer) as a fully integrated pharmaceutical company. We therefore determined that the individual multiplier for Messrs. Clark, Castelli, and Harford and
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Ms. Rosenberg, would range from 0-133% based on performance as described below. As noted above, the Compensation and Leadership Development Committee continues to believe that the bonus for the Chief Executive Officer should be determined solely by reference to the corporate multiplier. However, the Compensation and Leadership Development Committee periodically reviews and discusses its evaluation of the Chief Executive Officer’s performance and accomplishments in executive session, without the presence of the Chief Executive Officer, as part of its year-end executive officer review process.
The individual multiplier for each executive is determined after considering several factors, including achievement of individual objectives, departmental or organizational performance and other significant accomplishments. Individual objectives are necessarily tied to the particular area of expertise of the executive and are designed to support the Company’s achievement of its corporate goals. Individual goals are evaluated based on leadership and performance on specific functional goals that are tied to the corporate goals.
These objectives are set with the belief that full achievement will be difficult and challenging, but attainable, so long as the officer is fully committed to the accomplishment of such objectives through significant effort and dedication to the Company’s strategies and an ability to quickly adapt to a constantly evolving business environment.
Individual performance objectives of our named executive officers, other than the Chief Executive Officer, are determined by the Chief Executive Officer, to whom each named executive officer reports. During the annual review process, the Company’s Chief Executive Officer discusses with the Compensation and Leadership Development Committee his overall evaluation for each such executive, which includes the factors noted above. While the Compensation and Leadership Development Committee relies in part on the Chief Executive Officer’s evaluation of the other named executive officers, it also considers the degree of difficulty in attaining the Company’s goals and such executive’s accomplishments. In considering the degree of difficulty, the Compensation and Leadership Development Committee considers factors such as the influence of external events, including unanticipated clinical events and regulatory timelines, and the effort expended by executives. Upon the completion of such process, the Compensation and Leadership Development Committee determines the individual multiplier for each named executive officer, other than the Chief Executive Officer, based on the Compensation and Leadership Development Committee’s determination of such officer’s satisfaction of the applicable goals.
2025 Determinations
The Compensation and Leadership Committee believes that because of the Chief Executive Officer’s influence on the overall performance of Amicus, it is appropriate and in the best interests of our stockholders to base the Chief Executive Officer’s cash bonus solely on the achievement of the corporate objectives, without regard to an individual multiplier. For 2025, the Company’s corporate multiplier was determined to be 140%. In determining the individual multiplier for our named executive officers (excluding the Chief Executive Officer), the Compensation and Leadership Development Committee reviews the Chief Executive Officer’s recommendation as to each such executive officer’s individual and departmental performance throughout the year and how those performances supported the Company’s achievement of its corporate goals. The specific individual factors that the Compensation and Leadership Development Committee noted in determining each such named executive officer’s individual multiplier were as follows:
Simon Harford, Chief Financial Officer (120% Individual Multiplier)
●Achieved positive GAAP net income in the second half of 2025;
●Achieved positive net cash from operating activities in financial year 2025; and
●Managed budget and forecasting investment resource allocation to support revenue growth.
Ellen S. Rosenberg, Chief Legal Officer, and Corporate Secretary (120% Individual Multiplier)
●Led negotiations and completion of Dimerix License Agreement and BioMarin Merger Agreement;
●Led the Company’s global intellectual property litigation and settlement strategies;
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●Advised the Board of Directors and led the Board evaluation process; and
●Maintained SEC compliance in all of our activities.
David M. Clark, Chief People Officer (120% Individual Multiplier)
●Led the Human Resources strategies for the BioMarin Merger Agreement and interim operating covenants;
●Led the Company’s people and culture corporate goal achievement and searches for new members of the Company’s executive team;
●Led the information technology efforts to continue improving the Company’s cybersecurity, resulting in improved internal controls and NIST scores; and
●Successfully onboarded the Company's first Chief Corporate Affairs and Communications officer and built out the Company's communications and government and public affairs strategy.
Jeffrey P. Castelli, Chief Development Officer (125% Individual Multiplier)
●Led the R&D and Medical functions that achieved a number of key milestones including Galafold® and Pombiliti®+Opfolda® geographic expansion, label expansion, evidence generation and data dissemination;
●Contributed significantly to business development diligence resulting in acquisition of commercial rights to DMX-200 for FSGS in the United States; and
●Contributed significantly to successful Galafold® Hatch-Waxman litigation as inventor, witness and company representative.
Calculation of Annual Cash Incentive Bonuses
The calculation of the named executive officers’ individual cash incentive payments for service in 2025 is summarized in the table below.
Name and Principal Position | Corporate Multiplier (%) | Individual Multiplier (%) | Target Bonus (%) | Base Salary ($) | Payout ($) | ||||||||||||
Bradley L. Campbell | 140 | N/A | 75 | 825,000 | 866,250 | ||||||||||||
President and Chief Executive Officer | |||||||||||||||||
Simon Harford | 140 | 120 | 45 | 525,300 | 397,127 | ||||||||||||
Chief Financial Officer | |||||||||||||||||
Ellen S. Rosenberg | 140 | 120 | 45 | 535,600 | 404,914 | ||||||||||||
Chief Legal Officer and Corporate Secretary | |||||||||||||||||
David M. Clark | 140 | 120 | 45 | 497,948 | 376,449 | ||||||||||||
Chief People Officer | |||||||||||||||||
Jeffrey P. Castelli | 140 | 125 | 45 | 512,595 | 403,669 | ||||||||||||
Chief Development Officer | |||||||||||||||||
See "280G" Mitigation Actions" below for a description of when the 2025 cash incentives were paid to the named executive officers, and the terms of the clawback agreements they entered into with the Company in connection with the receipt of these payments.
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Long-Term Incentive Programs
We believe that long-term performance will be achieved through an ownership culture that rewards our named executive officers for maximizing stockholder value over time and that aligns the interests of our employees and management with those of stockholders. Our Amended and Restated 2007 Equity Incentive Plan, or the 2007 Plan, authorizes us to grant stock options, restricted stock, RSUs, PRSUs and other equity-based awards. . All of the equity grants that we made to our named executive officers in 2025 were granted under the 2007 Plan. At our 2025 Annual Meeting of Stockholders, our stockholders approved the Amicus Therapeutics. Inc. 2025 Equity Incentive Plan, or the 2025 Plan, to enable us to continue granting equity awards to our employees and other service providers. The 2025 Plan is an omnibus equity plan that allows for the grant of stock options, stock appreciation rights, restricted stock, RSUs (including PRSUs),stock grants, other stock-based awards and performance awards.
We continued our strategy for equity compensation in 2025 using a mix of non-qualified stock options, time based RSUs, and PRSUs for annual awards to our named executive officers. We utilize a value-based approach to allocate equity, with one third of the value assigned to each such type of equity vehicle in grants to each named executive officer. Our annual non-qualified stock option awards vest over a four-year period with 25% vesting one year after the vesting commencement date and the remainder vesting ratably each month thereafter over a three-year period, subject generally to continued service with the Company. The non-qualified stock options expire ten years after the date of grant. RSU awards vest, subject generally to continued service with the Company, over a four-year period, with 25% vesting each year upon the anniversary of the date of grant. PRSU awards vest over a three-year performance period and may be earned based on the attainment of the applicable goals at the end of such period, subject generally to continued service with the Company through the end of such period.
We use a mix of stock options, RSUs, and PRSUs as long-term incentive vehicles because we believe that:
●Stock options, RSUs and PRSUs, along with their vesting periods, provide a balanced mix to attract, motivate and retain executives;
●Stock options are inherently performance based. Because all of the value received by the recipient of a stock option is based on the growth of the stock price, stock options enhance the executives’ incentive to increase our stock price and maximize stockholder value;
●RSUs help enhance executive stock ownership while helping to retain executives. Final value depends on the stock price upon vesting;
●PRSUs align executives with the goals of the Company and its stockholders, while still assisting in the retention of our executives. Final value depends on company performance and the stock price upon vesting;
●Stock options, RSUs and PRSUs help to provide a balance to the overall executive compensation program as base salary and our annual performance bonus program focus on short-term compensation, while long-term incentives reward executives for increases in stockholder value over the longer term.
Stock Options, Restricted Stock Unit Awards, Performance Restricted Stock Unit Awards
The Compensation and Leadership Development Committee believes that granting annual equity awards provides management with a strong link to long-term corporate performance and the creation of stockholder value, as well as providing continued retention via long-term vesting. In 2025, the Compensation and Leadership Development Committee continued the approach of having such annual equity grants consist of stock options, RSUs and PRSUs (“LTI grants”).
The Compensation and Leadership Development Committee determines a dollar value for LTI grants to our named executive officers in its discretion, taking into account a number of factors which include the current price of our Common Stock, peer group executive compensation data provided by Pay Governance, each individual’s role and performance and recent Company developments. Specifically, the Compensation and Leadership Development Committee considered the following in determining the sizing of these equity grants for the Chief Executive Officer, and along with the Chief Executive Officer for the other executive officers: (i) relative contribution toward achievement of prior year corporate objectives, (ii) breadth of internal and external responsibilities, (iii) management responsibilities including managing direct reports, (iv) external benchmarking, and (v) tenure with Amicus. The specific individual factors that the Compensation and Leadership Development Committee
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relied on for granting each award are substantially similar to those factors that contributed to a determination of the individual multiplier for each named executive officer discussed above under “2025 Determinations.”
As described in the section entitled "Timing of Equity Awards" below, the Compensation and Leadership Development Committee approved a dollar value for LTI grants for the named executive officers shortly before the annual grant date of January 3, 2025. The approved amounts were:
Name and Principal Position | Approved Dollar Value ($) | ||||
Bradley L. Campbell President and Chief Executive Officer | $ | 7,700,000 | |||
Simon Harford Chief Financial Officer | 2,400,000 | ||||
Ellen S. Rosenberg Chief Legal Officer and Corporate Secretary | 3,250,000 | ||||
David M. Clark Chief People Officer | 2,000,000 | ||||
| Jeffrey P. Castelli Chief Development Officer | 2,400,000 | ||||
One third of the approved dollar value was then divided by the closing price of our stock on the established valuation date (December 27, 2024) to determine the number of RSUs granted; one third was divided by the closing price of our stock on the valuation date to determine the number of PRSUs granted (assuming target performance); and one third was divided by the Black-Scholes value of our stock on the valuation date to determine the number of stock options granted. The grant date fair values of the LTI grants are set forth below and reflected in the Summary Compensation Table and Grants of Plan Based Awards table. The grant date fair value for the PRSUs differs from the approved dollar value due to stock price fluctuations between the date upon which the number of shares underlying the PRSUs was set and the formal grant date of the PRSUs (June 3, 2025), which is when the PRSU goals were formally approved by the Compensation and Leadership Development Committee.
Name and Principal Position | Stock Options ($) | Restricted Stock Units ($) | Performance Restricted Stock Units ($) | Total ($) | ||||||||||
Bradley L. Campbell | 2,511,856 | 2,508,028 | 1,867,028 | 6,886,912 | ||||||||||
President and Chief Executive Officer | ||||||||||||||
Simon Harford | 782,917 | 781,717 | 581,926 | 2,146,560 | ||||||||||
Chief Financial Officer | ||||||||||||||
Ellen S. Rosenberg | 1,060,200 | 1,058,578 | 788,026 | 2,906,804 | ||||||||||
Chief Legal Officer and Corporate Secretary | ||||||||||||||
David M. Clark | 652,427 | 651,435 | 484,943 | 1,788,805 | ||||||||||
Chief People Officer | ||||||||||||||
| Jeffrey P. Castelli | 782,917 | 781,717 | 581,926 | 2,146,560 | ||||||||||
Chief Development Officer | ||||||||||||||
All of the stock option and RSU awards are subject to four-year ordinary course vesting schedules, while our PRSUs may be earned over a three-year performance period at 0% to 200% of target, based on the achievement of certain performance goals and subject generally to continued employment through the end of the performance period. At the time goals were established,
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the Compensation and Leadership Development Committee believed that these goals were difficult and challenging to attain and appropriately aligned incentives with performance. The performance goals for the PRSUs were weighted as follows:
2025 PRSU Performance Metrics and Weightings | ||||||||
TSR Goal (%) | Portfolio Goal (%) | Revenue Goal (%) | ||||||
| 50 | 25 | 25 | ||||||
The total stockholder return (“TSR”) goal compares the TSR of the Company’s common stock relative to the TSR of the Nasdaq Biotechnology Index (“NBI”) over the three-year performance period. Achievement of the 2025 PRSU TSR goal will be determined in accordance with the following schedule, with straight line interpolation applied for performance falling between such levels, provided that the payout is capped at 100% if the Company’s TSR is a negative number:
Performance Level | Three-Year TSR Ranking vs. NBI | Percentage of Granted TSR Shares to Vest | ||||||
Maximum | 90th Percentile or higher | 200% | ||||||
Above Target | 75th Percentile | 150% | ||||||
Target | 50th Percentile | 100% | ||||||
Threshold | 30th Percentile | 50% | ||||||
Below Threshold | Below 30th Percentile | 0% | ||||||
For confidential reasons, the Company does not generally disclose the specific revenue and portfolio performance goals associated with its PRSU grants until the end of the performance measurement period and level of achievement has been determined by the Compensation and Leadership Development Committee and approved by the Board.
Transaction Related Treatment
Treatment of Outstanding Stock Option Awards
All outstanding options (whether vested or unvested) will be cashed out upon the Transaction, provided that the strike price of the applicable option is lower than the per share transaction price. The cash payment for outstanding options will be calculated by deducting the strike price from the per share transaction price, multiplied by the number of options. Any options with strike prices equal to or above the per share transaction price will be cancelled for no value.
Treatment of Outstanding RSU Awards
Pursuant to the terms of the RSU award agreements, unvested RSUs will automatically vest upon the Transaction. See "280G Mitigation Actions" below for a description of the accelerated vesting treatment received by Messrs. Campbell and Harford for certain RSUs, and the terms of the clawback agreements they entered into with the Company in connection with the accelerated vesting treatment.
Treatment of Outstanding PRSU Awards
In 2023, 2024, and 2025 we granted PRSUs to our named executive officers which could be earned over a three-year performance period at 0% through 200% of target, based on the achievement of performance goals, and subject generally to continued employment through the end of the performance period. Historically, at the end of the performance period, the Compensation and Leadership Development Committee assessed the performance relative to each of the performance goals and determined whether and to what extent the applicable PRSUs are earned. Because the Merger Agreement provides for the cancellation and cash-out of all PRSU awards at closing, in late 2025 the performance goals for the outstanding 2023, 2024 and 2025 PRSU tranches were all evaluated based on accomplishments at the time of entry into the Merger Agreement and forecasted for the remainder of their respective measurement periods (with TSR attainment taking into account the price per share applicable to the Transaction). The Compensation and Leadership Development Committee recommended, and the Board
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subsequently agreed to, the following levels of achievement for each outstanding PRSU award (with payments to be made shortly following the closing of the Transaction):
Outstanding PRSU Award Performance (Relative to Target) | ||||||||
2023 (%) | 2024 (%) | 2025 (%) | ||||||
| 109.3 | 101.2 | 123.9 | ||||||
The table below reflects the total number of PRSUs each named executive officer stands to receive from their respective 2023, 2024, and 2025 PRSU grants upon closing of the Transaction:
Name and Principal Position | 2023 Target PRSUs | 2023 Earned PRSUs | 2024 Target PRSUs | 2024 Earned PRSUs | 2025 Target PRSUs | 2025 Earned PRSUs | ||||||||||||||
Bradley L. Campbell | 169,635 | 185,411 | 153,810 | 155,656 | 266,528 | 330,228 | ||||||||||||||
President and Chief Executive Officer | ||||||||||||||||||||
Simon Harford (1) | — | — | 57,392 | 58,081 | 83,073 | 102,927 | ||||||||||||||
Chief Financial Officer | ||||||||||||||||||||
Ellen S. Rosenberg | 77,749 | 84,980 | 63,131 | 63,889 | 112,495 | 139,381 | ||||||||||||||
Chief Legal Officer and Corporate Secretary | ||||||||||||||||||||
David M. Clark | 59,372 | 64,894 | 48,209 | 48,788 | 69,228 | 85,773 | ||||||||||||||
Chief People Officer | ||||||||||||||||||||
| Jeffrey P. Castelli | 70,681 | 77,254 | 57,392 | 58,081 | 83,073 | 102,927 | ||||||||||||||
Chief Development Officer | ||||||||||||||||||||
(1)Mr. Harford joined the Company in 2023 and thus did not receive a 2023 PRSU award.
280G Mitigation Actions
As described in the Merger Proxy Statement, the Company is permitted to implement certain strategies to mitigate excise taxes potentially resulting from the application of Sections 280G and 4999 of the Internal Revenue Code of 1986, as amended ("the Code") to change in control-related compensation (which would encompass Transaction-related compensation). In general and pursuant to the applicable tax rules, employees who have received higher W2 compensation in the calendar years prior to a change in control can have lesser (or no) excise taxes applied to change in control-related compensation.
Accordingly, the Compensation and Leadership Development Committee approved the following actions in December 2025:
2025 Bonus Acceleration: Each of the named executive officers received their 2025 bonuses in the amounts shown under the heading “Calculation of Annual Cash Incentive Bonus” in December 2025.
Accelerated RSU Vesting: Messrs. Campbell and Harford received the accelerated vesting of 212,323 RSUs, scheduled to originally vest by their terms between January 2, 2026 and March 15, 2026, and 144,486 RSUs, scheduled to originally vest by their terms between January 2, 2026 and January 3, 2028, respectively.
It is important to note that named executive officers who received the acceleration of their 2025 bonus payment or RSU vesting entered into a clawback agreement with the Company (the “Transaction Clawback Agreements”). Per the terms of the Transaction Clawback Agreements, each executive agreed to repay the accelerated 2025 bonus and value of the RSUs
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accelerated on their behalf, as applicable, should such individual’s employment with the Company terminate prior to the earlier of the consummation of the Transaction and the date the bonus or RSUs would have been payable or have vested, respectively, on their original terms.
Transaction Bonus Program
As described in the Merger Proxy Statement, the Company is permitted, and the Compensation and Leadership Development Committee approved, the establishment of a transaction bonus program, not to exceed $2,000,000 in the aggregate (the “Transaction Bonus Program”). Pursuant to the terms of the Transaction Bonus Program, all employees, including our named executive officers, except for Bradley Campbell, are eligible to participate and all awards granted thereunder would vest and become payable as of the closing date of the Transaction. No awards under the Transaction Bonus Program have been allocated as of February 12, 2026.
Non-Qualified Deferred Compensation Plans
Amicus Therapeutics, Inc. Restricted Stock Unit Deferral Plan
The Company maintains the Amicus Therapeutics, Inc. Restricted Stock Unit Deferral Plan, as amended (the “Stock Deferral Plan”). The Stock Deferral Plan provides eligible non-employee directors and executives, including each of the named executive officers, with the voluntary opportunity to defer the receipt of RSUs otherwise payable to such eligible executives to a distribution date elected by the participant. After a deferral election is made, a participant’s account is credited with the deferred RSUs. All RSUs deferred under the Stock Deferral Plan are fully vested. The Company does not otherwise contribute to the Stock Deferral Plan and the amount a participant receives at the end of a deferral period is based solely on the value of the Company’s stock at the end of the deferral period. Generally, a participant may voluntarily elect to re-defer any previously deferred RSUs for an additional period of not less than five years if, as required under the Code, such an election is made at least 12 months before the year in which the RSUs would otherwise be delivered.
Not only does the Stock Deferral Plan allow our eligible participants, including all of the named executive officers, to defer the federal income taxes otherwise payable upon the delivery of RSUs, but the Compensation and Leadership Development Committee believes that with respect to non-employee directors and executives who avail themselves of the deferral features of the Stock Deferral Plan, such persons will necessarily hold Company stock for a longer period of time. Accordingly, any RSUs deferred under the Stock Deferral Plan will continue to align such portion of our non-employee directors and named executive officers’ compensation with the interests of our stockholders for a longer period of time than would be provided by typical vesting periods. Regardless of a participant’s election, any deferred RSUs will be distributed following the non-employee director or executive’s death, disability, or separation of service from the Company.
In connection with the Transaction, each then outstanding RSU held by our directors and named executive officers pursuant to our Stock Deferral Plan will be cancelled and converted into the right of the holder to receive a cash payment (without interest and less any applicable tax withholdings) equal to the product of (i) $14.50 per share, multiplied by (ii) the number of shares of our common stock subject to such RSU, with payment to be made at the time provided for under the terms of the Restricted Stock Unit Deferral Plan. More information on the treatment of such RSUs in connection with the Transaction can be found in the Merger Proxy Statement.
Amicus Therapeutics, Inc. Cash Deferral Plan
The Company maintains the Amicus Therapeutics, Inc. Cash Deferral Plan, as amended (the “Cash Deferral Plan”). The Cash Deferral Plan provides eligible executives, including each of the named executive officers and non-employee directors, with the voluntary opportunity to defer receipt of such participant’s base salary, bonus and/or director’s fees, as applicable. Any such deferrals are credited to a bookkeeping account maintained for the participant. The participant may make periodic hypothetical investments of the account and gains and losses on such hypothetical investments will be credited to the participant’s account. A participant is fully vested in all amounts, including earnings deferred under the Cash Deferral Plan. Distribution of the deferred amounts will generally be made on the distribution date elected by the participant. Generally, a participant may voluntarily elect to re-defer any previously deferred amount for an additional period of not less than five years if, as required under the Code, such an election is made at least 12 months before the year in which the amount would otherwise be delivered. Regardless of a participant’s election, any deferred amount will be distributed following a change in control of the
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Company or upon the participant’s death, disability, or separation of service from the Company. The Company does not match any portion of participant deferrals in the Cash Deferral Plan.
All amounts deferred under the Cash Deferral Plan will continue for all purposes to be a part of the general funds of the Company and the amounts deferred by the participants, including all deemed gains and losses attributable thereto, will be subject to the claims of the general creditors of the Company in the event of the Company’s insolvency.
On July 29, 2025, the Board, upon the recommendation of the Compensation and Leadership Development Committee, elected to freeze the Cash Deferral Plan so that no new deferral elections could be made under the Cash Deferral Plan. However, any cash deferrals made pursuant to prior deferral elections remained outstanding.
Other Compensation
We maintain customary employee benefits that our named executive officers may participate in, including medical, dental, vision and life insurance coverage. All employees receive Company paid term life insurance equal to two times annual base salary, up to a maximum benefit of $1 million.
In addition, we provide a Company match for our 401(k) Plan, subject to federal guidelines and plan maximums. We match $1 for each $1 a participant, including each named executive officer, defers into the plan up to 5% of such participant’s salary and bonus paid during the year, subject to the IRS limit on eligible compensation. The match vests 100% on the participant’s one-year anniversary of employment at Amicus.
Furthermore, certain senior executives, including each named executive officer, are eligible to receive supplemental health benefits and financial consulting services. The value received from these benefits is calculated as imputed income and reflected in the “Summary Compensation Table” below.
Termination-Based Compensation
Upon termination of employment under certain circumstances, our named executive officers are entitled to receive varying types of compensation. Elements of this compensation may include payments based upon a number of months of base salary, bonus amounts, acceleration of vesting of equity, health care coverage and other similar benefits. We believe that our termination-based compensation and acceleration of vesting of equity arrangements are in line with severance packages offered to named executive officers of other similar companies based upon market information and are otherwise appropriate given the executive’s role and service to the Company. We also have granted severance and acceleration of vesting of equity benefits to our named executive officers under their employment agreements in the event of a change of control if the executive is terminated within a certain period of time following the change of control. We believe that change of control-related benefits are necessary in order for our named executive officers to direct their full attention to the successful consummation of a transaction without distraction. For more information on termination-based compensation see the section entitled “Severance Benefits and Change of Control Arrangements.”
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Stock Ownership Guidelines
We maintain stock ownership and retention guidelines for our directors and named executive officers to ensure that each of them has a long-term equity stake in Amicus, in order to both closely align the interests of directors and officers to those of our stockholders and to further our commitment to corporate governance.
Under the stock ownership guidelines, revised in 2022, named executive officers and directors must maintain stock ownership at a value equal to a multiple of their annual retainer or base salary, as applicable, as follows:
Position | Stock Retention Amount | ||||
Chief Executive Officer | 4 times executive’s base salary | ||||
Directors | 3 times director’s annual retainer | ||||
Other Executive Officers | 1 times executive’s base salary | ||||
Directors and executive officers have five years from the date they first became subject to the guidelines to attain the required stock ownership. Following the initial five year attainment period, stock ownership is recalculated every two years thereafter using the cash retainer or salary in effect at that time. Directors and executive officers then have those two additional years following each recalculation to acquire additional stock, as may be necessary, to satisfy the new stock ownership level and remain in compliance. Stock ownership that counts towards the requirement includes shares of Common Stock and any restricted stock units that were settled and deferred into the Stock Deferral Plan (as defined below). Stock options (vested or unvested), unvested restricted stock units and unearned performance awards are not included in determining compliance with these guidelines. All named executive officers and directors have met or are on track to meet the stock ownership guidelines within the requisite time period. The Compensation and Leadership Development Committee of the Board annually monitors compliance with this policy.
Prohibition on Hedging and Pledging
The Company considers it inappropriate for persons employed by or associated with the Company to engage in certain transactions related to the securities of the Company (“Subject Securities”) that could result in their interests no longer being aligned with the same interests and objectives as other stockholders of the Company. Therefore, as part of its anti-hedging and anti-pledging policy, the Company restricts these persons from hedging, engaging in short sales, transacting in publicly traded options, and pledging Subject Securities.
Certain hedging and monetization transactions involve the establishment of a short position in the Subject Securities and limit or eliminate a person’s ability to profit from an increase in the value of the Subject Securities. Accordingly, these transactions can cause a person’s interests to be misaligned with other stockholders of the Company. The Company therefore prohibits its directors, executive officers, and employees from engaging in any hedging and monetization transactions involving the Subject Securities. The Company’s directors and executive officers are also prohibited from engaging in short sales of Subject Securities (sales of securities that are not then owned).
Subject Securities held in a margin account or pledged as collateral for a loan may be sold without a person’s consent if he or she fails to meet a margin call or defaults on a loan, which may occur at a time when the covered person is aware of material nonpublic information or is otherwise not permitted to trade in Company securities. Therefore, our directors, executive officers and employees are prohibited from engaging in these activities.
Insider Trading Policy
We have adopted insider trading policies and procedures applicable to our employees, executive officers, directors and the Company that we believe are reasonably designed to promote compliance with insider trading laws, rules and regulations and Nasdaq listing standards applicable to us (the “Insider Trading Policy”).
Our Policy prohibits executive officers, directors, employees, agents and contractors (“Covered Persons”) of the Company from trading in the Company's securities while in possession of material non-public information. Our Policy restricts Covered Persons from trading in the Company's securities during certain periods and requires pre-clearance for their trades in the
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Company's securities from the Company's legal department. Our Insider Trading Policy was filed as exhibit 19.1 on this Form 10-K.
Clawback Policy
We have also historically maintained a policy for recoupment (or “clawback”) of performance-based compensation in the event of a financial restatement. On November 2nd, 2023, effective as of October 2nd 2023, our Board adopted a new clawback policy (the “Clawback Policy”) that fully aligns to the Nasdaq listing standards adopted in accordance with Section 10D of the Exchange Act, which govern such policies. A copy of our Clawback Policy is publicly available as an exhibit to the Company’s most recently filed Form 10-K.
Our Clawback Policy covers current and former executive officers, including the principal accounting officer, and applies to any compensation granted, earned, or vested, based wholly or in part on the attainment of a financial reporting measure, including non-GAAP, stock price or total stockholder return metrics. In the event of a financial restatement, the policy dictates that the Company must recoup the incremental amount an executive officer erroneously received as a result of the misstated financials, regardless of whether or not executive misconduct was present. The look-back period for recoupment consists of the three completed fiscal years preceding the date upon which the restatement is deemed required under the rules. The Compensation and Leadership Development Committee has broad discretion in determining the means of recovery, provided such approach is permitted under the Nasdaq rules and done reasonably promptly.
Timing of Equity Awards
We did not approve annual equity awards to our employees for 2026 in light of the Transaction but rather elected to provide cash awards instead, with the cash awards valued at 25% of the annual long-term incentive grant each employee was slated to receive in 2026. These cash awards in lieu of equity will be paid upon the closing of the Transaction, provided such applicable employee remains with the Company through the closing date.
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COMPENSATION AND LEADERSHIP DEVELOPMENT COMMITTEE REPORT
The Compensation and Leadership Development Committee is comprised entirely of independent directors. The Compensation and Leadership Development Committee of our Board has reviewed and discussed the Compensation Discussion and Analysis required by Item 402(b) of Regulation S-K, with our management. Based on this review and discussion, the Compensation and Leadership Development Committee has recommended to the Board that the Compensation Discussion and Analysis be included in this Form 10-K.
Members of the Amicus Therapeutics, Inc.
Compensation and Leadership Development Committee:
Margaret G. McGlynn, Chair
Lynn D. Bleil
Eiry W. Roberts, M.D.
Glenn P. Sblendorio
Notwithstanding anything to the contrary set forth in any of our previous or future filings under the Securities Act of 1933, as amended (the “Securities Act”) or the Exchange Act that might incorporate this Form 10-K or future filings with the SEC, in whole or in part, the above report shall not be deemed to be “soliciting material” or “filed” with the SEC and shall not be deemed to be incorporated by reference into any such filing.
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Executive Compensation
SUMMARY COMPENSATION TABLE
The following table provides information regarding the compensation that we paid to each person serving as our principal chief executive officer, our principal financial officer, and our three other most highly compensated executive officers (collectively, the “named executive officers”).
Name and Principal Position | Fiscal Year | Salary ($) | Bonus(1) ($) | Stock Awards(2) ($) | Option Awards(2) ($) | Change in Pension Value & Non- Qualified Deferred Compens-ation Earnings ($) | All Other Compensation ($) | Total ($) | ||||||||||||||||||
Bradley L. Campbell | 2025 | 822,885 | 866,250 | 4,700,158(8) | 2,511,856 | — | 45,783(3) | 8,946,931 | ||||||||||||||||||
President and Chief | 2024 | 767,308 | 623,700 | 4,990,365 | 2,105,627 | — | 39,943 | 8,526,943 | ||||||||||||||||||
Executive Officer | 2023 | 698,558 | 706,650 | 4,734,510 | 2,023,673 | — | 38,975 | 8,202,366 | ||||||||||||||||||
Simon Harford | 2025 | 524,904 | 397,127 | 2,571,355(9) | 782,917 | — | 13,170(4) | 4,289,473 | ||||||||||||||||||
Chief Financial | 2024 | 514,423 | 275,319 | 1,862,083 | 785,678 | — | 11,562 | 3,449,065 | ||||||||||||||||||
Officer | 2023 | 173,077 | 111,045 | 1,349,999 | 1,346,048 | — | 416 | 2,980,585 | ||||||||||||||||||
Ellen S. Rosenberg | 2025 | 534,808 | 404,914 | 1,846,604 | 1,060,200 | — | 45,898(5) | 3,892,424 | ||||||||||||||||||
Chief Legal Officer and | 2024 | 514,423 | 332,886 | 2,048,280 | 864,243 | — | 40,475 | 3,800,307 | ||||||||||||||||||
Corporate Secretary | 2023 | 499,704 | 363,420 | 2,169,972 | 927,515 | 81,376 | 33,170 | 4,075,157 | ||||||||||||||||||
David M. Clark | 2025 | 497,390 | 376,449 | 1,136,378 | 652,427 | — | 43,581(6) | 2,706,225 | ||||||||||||||||||
Chief People Officer | 2024 | 482,903 | 270,197 | 1,564,136 | 659,967 | — | 38,006 | 3,015,209 | ||||||||||||||||||
2023 | 469,059 | 341,153 | 1,657,073 | 708,284 | — | 39,030 | 3,214,599 | |||||||||||||||||||
Jeffrey P. Castelli | 2025 | 511,837 | 403,669 | 1,363,643 | 782,917 | — | 45,406(7) | 3,107,472 | ||||||||||||||||||
Chief Development | 2024 | 491,807 | 263,494 | 1,862,083 | 785,678 | — | 39,834 | 3,442,896 | ||||||||||||||||||
Officer | 2023 | 464,721 | 352,049 | 1,972,703 | 843,196 | — | 39,093 | 3,671,762 | ||||||||||||||||||
(1)The 2025 amount represents bonuses earned with respect to the 2025 performance year, which were paid in 2025.
(2)The grant date fair value of time-based restricted stock unit awards (“RSUs”), performance based restricted stock unit awards (“PRSUs”) and option awards granted to our named executive officers was computed in accordance with FASB ASC Topic 718, Compensation—Stock Compensation. Assumptions made in this valuation are discussed in Note 9 — Stock-based Compensation. These amounts reflect the stock price at the time of the grant. In accordance with SEC rules, the amounts reported in the Stock Awards column for 2025 include the grant date fair value of the RSUs and PRSUs granted during 2025. The following table provides information regarding the 2025 PRSUs based on the expected performance outcomes (and is the grant date fair value of the award, as reflected in the Summary Compensation Table) and maximum performance outcomes:
Name | Grant Date Fair Value for 2025 PRSUs (i.e., Based on Expected Performance) ($) | Value at Grant Date Assuming Maximum Performance ($) | ||||||
Bradley L. Campbell | 1,867,028 | 3,734,056 | ||||||
Simon Harford | 581,926 | 1,163,852 | ||||||
Ellen S. Rosenberg | 788,026 | 1,576,052 | ||||||
David M. Clark | 484,942 | 969,884 | ||||||
Jeffrey P. Castelli | 581,926 | 1,163,852 | ||||||
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(3)Includes $17,135 of 401(k) employer match, $825 for health care savings account, $1,248 in life insurance premiums, $4,500 for executive health benefits received, and $22,075 for financial consulting services.
(4)Includes $12,300 of 401(k) employer match and $870 in life insurance premiums.
(5)Includes $17,250 of 401(k) employer match, $825 for health care savings account, $1,248 in life insurance premiums, $4,500 for executive health benefits received, and $22,075 for financial consulting services.
(6)Includes $14,938 of 401(k) employer match, $825 for health care savings account, $1,243 in life insurance premiums, $4,500 for executive health benefits received, and $22,075 for financial consulting services.
(7)Includes $16,758 of 401(k) employer match, $825 for health care savings account, $1,248 in life insurance premiums, $4,500 for executive health benefits received, and $22,075 for financial consulting services.
(8)Includes $325,102 of value associated with awards expected to vest in 2026 but accelerated to vest in 2025.
(9)Includes $534,525 of value associated with awards expected to vest in 2026, $534,525 of awards expected to vest in 2027, and $138,662 of awards expected to vest in 2028 but accelerated to vest in 2025.
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Grants of Plan Based Awards
The following table presents information concerning grants of equity awards to each of the named executive officers during 2025.
Estimated Future Payouts Under Equity Incentive Plan Awards(1) | All Other Stock Awards: Number of Shares of RSUs (2)(#) | All Other Option Awards: Number of Securities Underlying Options (3)(#) | Exercise or Base Price of Option Awards ($/Sh) | Grant Date Fair Value of Stock and Option Awards (4)($) | |||||||||||||||||||||||||
Name | Grant Date | Approval Date | Threshold (#) | Target (#) | Maximum (#) | ||||||||||||||||||||||||
Bradley L. | 1/3/2025 | 1/3/2025 | 482,908 | 9.41 | 2,511,856 | ||||||||||||||||||||||||
Campbell | 1/3/2025 | 1/3/2025 | 266,528 | 2,508,028 | |||||||||||||||||||||||||
President and | 6/3/2025(5) | 1/3/2025 | 66,632 | 133,264 | 266,528 | 1,032,796 | |||||||||||||||||||||||
Chief Executive | 6/3/2025(5) | 1/3/2025 | 33,316 | 66,632 | 133,264 | 417,116 | |||||||||||||||||||||||
Officer | 6/3/2025(5) | 1/3/2025 | 33,316 | 66,632 | 133,264 | 417,116 | |||||||||||||||||||||||
Simon Harford | 1/3/2025 | 1/3/2025 | 150,517 | 9.41 | 782,917 | ||||||||||||||||||||||||
Chief Financial | 1/3/2025 | 1/3/2025 | 83,073 | 781,717 | |||||||||||||||||||||||||
Officer | 6/3/2025(5) | 1/3/2025 | 20,768 | 41,536 | 83,072 | 321,904 | |||||||||||||||||||||||
6/3/2025(5) | 1/3/2025 | 10,385 | 20,769 | 41,538 | 130,014 | ||||||||||||||||||||||||
6/3/2025(5) | 1/3/2025 | 10,384 | 20,768 | 41,536 | 130,008 | ||||||||||||||||||||||||
Ellen Rosenberg | 1/3/2025 | 1/3/2025 | 203,825 | 9.41 | 1,060,200 | ||||||||||||||||||||||||
Chief Legal | 1/3/2025 | 1/3/2025 | 112,495 | 1,058,758 | |||||||||||||||||||||||||
Officer and | 6/3/2025(5) | 1/3/2025 | 28,124 | 56,247 | 112,494 | 435,914 | |||||||||||||||||||||||
Corporate | 6/3/2025(5) | 1/3/2025 | 14,062 | 28,124 | 56,248 | 176,056 | |||||||||||||||||||||||
Secretary | 6/3/2025(5) | 1/3/2025 | 14,062 | 28,124 | 56,248 | 176,056 | |||||||||||||||||||||||
David M. Clark | 1/3/2025 | 1/3/2025 | 125,430 | 9.41 | 652,427 | ||||||||||||||||||||||||
Chief People | 1/3/2025 | 1/3/2025 | 69,228 | 651,435 | |||||||||||||||||||||||||
Officer | 6/3/2025(5) | 1/3/2025 | 17,307 | 34,614 | 69,228 | 268,259 | |||||||||||||||||||||||
6/3/2025(5) | 1/3/2025 | 8,654 | 17,307 | 34,614 | 108,342 | ||||||||||||||||||||||||
6/3/2025(5) | 1/3/2025 | 8,654 | 17,307 | 34,614 | 108,342 | ||||||||||||||||||||||||
Jeffrey P. Castelli | 1/3/2025 | 1/3/2025 | 150,517 | 9.41 | 782,917 | ||||||||||||||||||||||||
Chief | 1/3/2025 | 1/3/2025 | 83,073 | 781,717 | |||||||||||||||||||||||||
Development | 6/3/2025(5) | 1/3/2025 | 20,768 | 41,536 | 83,072 | 321,904 | |||||||||||||||||||||||
Officer | 6/3/2025(5) | 1/3/2025 | 10,385 | 20,769 | 41,538 | 130,014 | |||||||||||||||||||||||
6/3/2025(5) | 1/3/2025 | 10,384 | 20,768 | 41,536 | 130,008 | ||||||||||||||||||||||||
(1)Amounts represent PRSUs granted to named executive officers during the 2025 fiscal year. As of the time of the grant, the criteria that was established to determine the number of PRSUs that could be earned by each named executive officer is described above under “Performance-Based Restricted Stock Units”. For each named executive officer, the first row of PRSUs shown in the table was tied to the TSR goal, the second row the portfolio goal and the third row the revenue goal. Pursuant to the terms of the grants, the named executive officers must generally be in continuous service with the Company through December 31, 2027 to vest into the PRSUs. However, see "Transaction Related Treatment" above for a description of the treatment of the PRSUs in connection with the Transaction.
(2)Vesting of the RSU is generally subject to the participant’s continuous service with the Company through the applicable vesting date with the following schedule: 25% of the total number of shares vest on the first anniversary of the grant date (the “vesting commencement date”), with 25% on each of the next three successive vesting commencement date anniversaries thereafter. However, see "Transaction Related Treatment" above for a description of the treatment of the RSUs in connection with the Transaction.
(3)Each option has a term of ten years and vests in accordance with the following schedule: 25% of the total number of shares vest on the first anniversary of the grant date and 1/36th of the remaining number of shares
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vest on the first day of each of the following 36 months. The exercise price for our option grants is equal to the closing stock price on the grant date. However, see "Transaction Related Treatment" above for a description of the treatment of the stock options in connection with the Transaction.
(4)Amounts represent the grant date fair value calculated in accordance with FASB ASC 718, as stated in footnote #2 to the Summary Compensation Table.
(5)In January 2025, the Compensation and Leadership Development Committee determined the number of PRSUs each executive officer would receive in connection with their 2025 annual grant. The PRSU goals, and their relative percentages, were approved by the Committee on June 3, 2025 and were valued as of such date.
Outstanding Equity Awards at Fiscal Year-End
The following table presents the outstanding equity awards held by each of the named executive officers as of December 31, 2025.
Option Awards | Stock Awards | ||||||||||||||||||||||||||||
Name and Principal Position | Number of Securities Underlying Unexercised Options (#) Exercisable | Number of Securities Underlying Unexercised Options (#) Unexercisable (1) | Option Exercise Price ($) | Option Expiration Date | Number of Units of Stock That Have Not Vested (#)(2) | Market Value of Units of Stock That Have Not Vested ($)(3) | Equity Incentive Plan Awards: Number of Unearned Units of Stock That Have Not Vested (#)(4) | Equity Incentive Plan Awards: Market Value of Unearned Units of Stock That Have Not Vested ($)(3) | |||||||||||||||||||||
Bradley L. Campbell President and Chief Executive Officer | 75,000 | — | 6.10 | 6/15/2026 | 42,411(7) | 603,933 | |||||||||||||||||||||||
| 103,578 | — | 5.13 | 1/3/2027 | 76,906(8) | 1,095,141 | ||||||||||||||||||||||||
| 56,329 | — | 15.67 | 1/3/2028 | 199,896(9) | 2,846,519 | ||||||||||||||||||||||||
| 151,515 | — | 10.04 | 1/2/2029 | 27,811(10) | 396,034 | ||||||||||||||||||||||||
| 188,257 | — | 9.55 | 1/2/2030 | 9,272(10) | 132,032 | ||||||||||||||||||||||||
| 92,792 | — | 21.78 | 1/4/2031 | 55,623(10) | 792,068 | ||||||||||||||||||||||||
| 277,633 | 5,922(5) | 12.11 | 1/3/2032 | 92,705(10) | 1,320,119 | ||||||||||||||||||||||||
| 176,220 | 11,750(6) | 8.95 | 3/15/2032 | 31,131(11) | 443,307 | ||||||||||||||||||||||||
| 219,556 | 81,553(7) | 11.93 | 1/3/2033 | 46,697(11) | 664,961 | ||||||||||||||||||||||||
| 127,220 | 138,297(8) | 14.24 | 1/2/2034 | 77,828(11) | 1,108,269 | ||||||||||||||||||||||||
| — | 482,908(9) | 9.41 | 1/3/2035 | 82,55712) | 1,175,612 | ||||||||||||||||||||||||
82,557(12) | 1,175,612 | ||||||||||||||||||||||||||||
165,114(12) | 2,351,225 | ||||||||||||||||||||||||||||
Simon Harford Chief Financial Officer | 110,268 | 78,775(13) | 12.62 | 8/21/2033 | 14,348(8) | 204,316 | |||||||||||||||||||||||
| 47,472 | 51,601(8) | 14.24 | 1/2/2034 | 20,768(9) | 295,751 | ||||||||||||||||||||||||
| — | 150,517 | 9.41 | 1/3/2035 | 11,617(11) | 165,422 | ||||||||||||||||||||||||
17,424(11) | 248,112 | ||||||||||||||||||||||||||||
29,040(11) | 413,535 | ||||||||||||||||||||||||||||
25,732(12) | 366,417 | ||||||||||||||||||||||||||||
25,733(12) | 366,435 | ||||||||||||||||||||||||||||
51,463(12) | 732,835 | ||||||||||||||||||||||||||||
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Option Awards | Stock Awards | ||||||||||||||||||||||||||||
Name and Principal Position | Number of Securities Underlying Unexercised Options (#) Exercisable | Number of Securities Underlying Unexercised Options (#) Unexercisable (1) | Option Exercise Price ($) | Option Expiration Date | Number of Units of Stock That Have Not Vested (#)(2) | Market Value of Units of Stock That Have Not Vested ($)(3) | Equity Incentive Plan Awards: Number of Unearned Units of Stock That Have Not Vested (#)(4) | Equity Incentive Plan Awards: Market Value of Unearned Units of Stock That Have Not Vested ($)(3) | |||||||||||||||||||||
Ellen S. Rosenberg Chief Legal Officer and Corporate Secretary | 39,762 | — | 15.67 | 1/3/2028 | 18,284(5) | 260,364 | |||||||||||||||||||||||
| 93,663 | — | 10.04 | 1/2/2029 | 38,875(7) | 553,580 | ||||||||||||||||||||||||
| 107,575 | — | 9.55 | 1/2/2030 | 47,349(8) | 674,250 | ||||||||||||||||||||||||
| 53,355 | — | 21.78 | 1/4/2031 | 112,495(9) | 1,601,929 | ||||||||||||||||||||||||
| 131,231 | 2,813(5) | 12.11 | 1/3/2032 | 12,747(10) | 181,511 | ||||||||||||||||||||||||
| 100,627 | 37,381(7) | 11.93 | 1/3/2033 | 4,251(10) | 60,530 | ||||||||||||||||||||||||
| 52,215 | 56,765(8) | 14.24 | 1/2/2034 | 25,493(10) | 363,022 | ||||||||||||||||||||||||
| — | 203,825(9) | 9.41 | 1/3/2035 | 42,489(10) | 605,047 | ||||||||||||||||||||||||
12,779(11) | 181,966 | ||||||||||||||||||||||||||||
19,166(11) | 272,928 | ||||||||||||||||||||||||||||
31,944(11) | 454,879 | ||||||||||||||||||||||||||||
34,846(12) | 496,202 | ||||||||||||||||||||||||||||
34,846(12) | 496,202 | ||||||||||||||||||||||||||||
69,690(12) | 992,386 | ||||||||||||||||||||||||||||
David M. Clark Chief People Officer | 84,842 | — | 12.84 | 10/9/2028 | 14,770(13) | 210,325 | |||||||||||||||||||||||
| 77,134 | — | 10.04 | 1/2/2029 | 29,686(7) | 422,729 | ||||||||||||||||||||||||
| 91,439 | — | 9.55 | 1/2/2030 | 36,157(8) | 514,876 | ||||||||||||||||||||||||
| 44,076 | — | 21.78 | 1/4/2031 | 69,228(9) | 985,807 | ||||||||||||||||||||||||
| 105,991 | 2,275(14) | 12.11 | 1/14/2032 | 9,733(10) | 138,600 | ||||||||||||||||||||||||
| 76,832 | 28,556(7) | 11.93 | 1/3/2033 | 3,246(10) | 46,226 | ||||||||||||||||||||||||
| 39,868 | 43,353(8) | 14.24 | 1/2/2034 | 19,467(10) | 277,216 | ||||||||||||||||||||||||
| — | 125,430(9) | 9.41 | 1/3/2035 | 32,447(10) | 462,042 | ||||||||||||||||||||||||
9,759(11) | 138,964 | ||||||||||||||||||||||||||||
14,636(11) | 208,410 | ||||||||||||||||||||||||||||
24,393(11) | 347,360 | ||||||||||||||||||||||||||||
21,443(12) | 305,354 | ||||||||||||||||||||||||||||
21,443(12) | 305,354 | ||||||||||||||||||||||||||||
42,887(12) | 610,707 | ||||||||||||||||||||||||||||
Jeffrey P. Castelli Chief Development Officer | 39,762 | — | 15.67 | 1/3/2028 | 14,770(5) | 210,325 | |||||||||||||||||||||||
| 82,644 | — | 10.04 | 1/2/2029 | 35,341(7) | 503,256 | ||||||||||||||||||||||||
| 107,575 | — | 9.55 | 1/2/2030 | 43,044(8) | 612,947 | ||||||||||||||||||||||||
| 46,396 | — | 21.78 | 1/4/2031 | 83,073(9) | 1,182,960 | ||||||||||||||||||||||||
| 105,991 | 2,275(5) | 12.11 | 1/3/2032 | ||||||||||||||||||||||||||
| 91,464 | 33,998(7) | 11.93 | 1/3/2033 | 11,558(10) | 165,013 | ||||||||||||||||||||||||
| 47,472 | 51,601(8) | 14.24 | 1/2/2034 | 3,864(10) | 55,020 | ||||||||||||||||||||||||
| — | 150,517(9) | 9.41 | 1/3/2035 | 23,176(10) | 330,026 | ||||||||||||||||||||||||
38,627(10) | 550,043 | ||||||||||||||||||||||||||||
11,617(11) | 165,422 | ||||||||||||||||||||||||||||
17,424(11) | 248,112 | ||||||||||||||||||||||||||||
29,040(11) | 413,535 | ||||||||||||||||||||||||||||
25,732(12) | 366,417 | ||||||||||||||||||||||||||||
25,733(12) | 366,435 | ||||||||||||||||||||||||||||
51,463(12) | 732,835 | ||||||||||||||||||||||||||||
(1)Unless otherwise indicated, 25% of the total number of shares subject to the option vest on the first anniversary of the date of grant; the remainder vest 1/36th per month thereafter, subject generally to the participant’s continuous service with the Company through the applicable vesting date. However, see "Transaction Related Treatment" above for a description of the treatment of stock options in connection with the Transaction.
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(2)Vesting of the RSUs is subject generally to the participant’s continuous service with the Company through the applicable vesting date with the following schedule: unless otherwise indicated, 25% of the total number of shares vest on the first anniversary of the grant date, with 25% on each successive grant date anniversary for the next three years. However, see "Transaction Related Treatment" above for a description of the treatment of the RSUs in connection with the Transaction.
(3)The market value is based on the closing stock price of $14.24 on December 31, 2025.
(4)Vesting of PRSUs are generally subject to the attainment of performance goals applicable to such awards and the participant’s continuous service with the Company. The amounts shown in the table above assumes performance at the forecasted level for all outstanding PRSUs. Please see "Transaction Related Treatment" above for a description of the treatment of the PRSUs in connection with the Transaction.
(5)The date of grant was January 3, 2022.
(6)The date of grant was March 15, 2022.
(7)The date of grant was January 3, 2023.
(8)The date of grant was January 2, 2024.
(9)The date of grant was January 3, 2025.
(10)Subject generally to the participant’s continued service and the attainment of the applicable performance goals, these PRSUs vested on December 31, 2025 and are displayed at the forecasted performance at closing of 109.3% as previously disclosed in the Merger Proxy statement.
(11)Subject generally to the participant’s continued service and the attainment of the applicable performance goals, these PRSUs vest on December 31, 2026 and are displayed at the forecasted performance at closing of 101.2% as previously disclosed in the Merger Proxy Statement .
(12)Subject generally to the participant’s continued service and the attainment of the applicable performance goals, these PRSUs vest on December 31, 2027 and are displayed at the forecasted performance at closing of 123.9% as previously disclosed in the Merger Proxy Statement.
(13)The date of grant was August 21, 2023.
(14)The date of grant was January 14, 2022, but is deemed to be January 3, 2022 for vesting purposes.
Option Exercises and Stock Vested at Year End
Our executive officers must use pre-established trading plans to sell shares of Amicus Therapeutics, Inc. stock. Such trading plans must conform with Rule 10b5-1, meaning they may only be entered into during an open trading window and when the executive officer is not in possession of material non-public information about the Company. Further, we enforce the statutorily required waiting period following the establishment of a trading plan before any trades may be executed to ensure compliance with rule 10b5-1 affirmative defense requirements. Our policy is designed to provide safeguards that will allow our executives an opportunity to realize the value intended by the Company in granting equity-based awards.
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The following table shows information regarding option exercises and stock vested for each named executive officer during the year ended December 31, 2025.
Option Awards | Stock Awards | |||||||||||||
Name and Principal Position | Number of Shares Acquired on Exercise (#) | Value Realized upon Exercise(1) ($) | Number of Shares Acquired on Vesting(2) (#) | Value Realized upon Vesting(3) ($) | ||||||||||
Bradley L. Campbell | 100,000(4) | 157,043 | 372,417 | 4,514,915 | ||||||||||
President and Chief Executive Officer | ||||||||||||||
Simon Harford | — | — | 185,578 | 2,395,766 | ||||||||||
Chief Financial Officer | ||||||||||||||
Ellen S. Rosenberg | 95,621(5) | 443,694 | 61,788 | 579,847 | ||||||||||
Chief Legal Officer and Corporate Secretary | ||||||||||||||
David M. Clark | — | — | 48,505 | 455,227 | ||||||||||
Chief People Officer | ||||||||||||||
Jeffrey P. Castelli | 50,000(4) | 60,575 | 53,989 | 506,602 | ||||||||||
Chief Development Officer | ||||||||||||||
(1)The value realized is the difference between the fair market value of a share of our Common Stock at the time of exercise and the option exercise price, multiplied by the number of shares acquired upon each exercise.
(2)Reflects RSUs that vested during 2025.
(3)The value realized on vesting of stock awards is based on the closing price of the Company’s common stock on the date of vesting.
(4)These options were scheduled to expire on January 4, 2026.
(5)These options were scheduled to expire on June 15, 2026 and January 3, 2027.
Non-Qualified Deferred Compensation
As described more fully above, our Cash Deferral Plan and Stock Deferral Plan (collectively, the “Deferral Plans”) cover our executive officers and non-employee directors. Earnings are determined solely by a participant’s hypothetical investment of any amount deferred in any pre-selected investment permitted under the Cash Deferral Plan or in the value of our stock, with respect to the Stock Deferral Plan. All amounts in the Deferral Plans are fully vested at all times. No named executive officer participates in the Deferral Plans except for Ms. Rosenberg, who participates in the Stock Deferral Plan. The details of the change in value from Ms. Rosenberg's participation in the Stock Deferral Plan is described below.
Name and Principal Position | Executive Contributions in Last Fiscal Year ($) | Aggregate Earnings in Last Fiscal Year ($) | Aggregate Distributions in Last Fiscal Year ($) | Aggregate Balance at Last Fiscal Year(1) ($) | ||||||||||
Ellen S. Rosenberg | — | 198,097(2) | — | 585,250 | ||||||||||
Chief Legal Officer and Corporate Secretary | ||||||||||||||
(1)A total of $641,349 was previously reported as compensation to Ms. Rosenberg in our Summary Compensation Table for previous years. Because the “Aggregate Balance at Last Fiscal Year” depends upon the value of our stock price, which fluctuates over time, this may be less or more than the value of the historical contributions that Ms. Rosenberg has made to the plan.
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(2)The gain represents the increase in value during 2025 of shares subject to RSUs deferred under the Stock Deferral Plan.
Severance Benefits and Change of Control Arrangements
The named executive officers are parties to employment agreements which set forth certain severance and change in control benefits, as described below.
NEO Employment Agreements
Bradley L. Campbell
We employ Mr. Campbell as our Chief Executive Officer pursuant to an employment agreement. The agreement continues until either Mr. Campbell or the Company provide written notice of termination to the other in accordance with the terms of the agreement. Upon the termination of his employment by the Company other than for cause and not in connection with a change in control event, Mr. Campbell has the right to receive (i) a severance payment in an amount equal to his then current base salary payable over 18 months in accordance with the Company’s regular payroll practices, (ii) an additional payment equal to 150% of the target bonus for the year in which the termination occurs, and (iii) continuation of health care coverage with full premiums to be paid by the Company for up to 18 months. Further, the vesting of all options and RSUs then held by Mr. Campbell shall accelerate by 18 months and any unvested PRSUs held by Mr. Campbell shall also accelerate, such that the portion of those PRSUs otherwise scheduled to vest during the 18-month period immediately following such separation from service will become vested. Mr. Campbell is not entitled to severance payments if the Company terminates him for cause or if he resigns independent of a change in control event.
Further, if upon the termination of Mr. Campbell’s employment by the Company other than for cause, or if Mr. Campbell resigns for good reason, in each case within twelve months following a change of control in the Company, then Mr. Campbell has the right to receive (i) a severance payment in an amount equal to two times his then current base salary payable over 24 months in accordance with our regular payroll practices, (ii) an additional payment equal to 200% of the target bonus for the year in which the termination occurs, and (iii) continuation of health care coverage with premiums to be paid by the Company for up to 24 months. Further, the vesting of all remaining unvested options and restricted stock grants then held by Mr. Campbell would accelerate in full.
Finally, if Mr. Campbell’s employment ceases due to an inability to work, he will be entitled to the continuation of health care coverage with full premiums to be paid by the Company for up to 12 months. We believe that the severance package for our Chief Executive Officer is appropriate considering his role, responsibilities, and his excellent historical and continued service to the Company.
Other Named Executive Officers
Messrs. Clark, Castelli and Harford and Ms. Rosenberg. We employ Mr. Clark as our Chief People Officer, Dr. Castelli as our Chief Development Officer, Mr. Harford as our Chief Financial Officer and Ms. Rosenberg as our Chief Legal Officer and Corporate Secretary, pursuant to their respective employment agreements. If any of these executive officers is terminated without cause (other than within 12 months following a change in control), then the executive officer has the right to receive the following:
●continuation of such executive’s base salary for 12 months;
●an amount equal to the target bonus for such executive officer pro-rated for the number of months actually worked in the year of termination;
●accelerated vesting of equity awards otherwise scheduled to vest within twelve months; and
●continuation of health care coverage with full premiums to be paid by the Company for a period of 12 months.
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In addition, if any of the executive officers are terminated other than for cause within 12 months following a change of control or, if within 12 months following a change of control, the executive officer resigns for good reason, then the executive officer has the right to receive:
●continuation of such executive’s base salary for 18 months in an amount equal to such executive’s then current base salary;
●an amount equal to such executive officer’s target annual bonus;
●any outstanding equity awards held by the executive officer will vest (with PRSUs vesting at target or such greater level as determined by the Board); and
●continuation of health care coverage with full premiums to be paid by the Company for a period of 18 months.
Finally, if any of the executive officers’ employment ceases due to death or disability, such executive will be entitled to continuation of health care coverage with full premiums to be paid by the Company for up to 12 months.
As a condition to the payment of the foregoing severance benefits, a departing executive officer is required to execute a general release of claims against the Company and its affiliates. Each named executive officer is bound by non-disclosure, inventions transfer, non-solicitation and non-competition covenants that prohibit the executive officer from competing with the Company during the term of his or her employment and for twelve months after termination of employment.
Equity Award Agreements
On June 5, 2025, the Board determined that the treatment of equity awards granted under the Amicus Amended and Restated 2007 Equity Incentive Plan upon a participant's retirement, disability or death would generally follow the treatment approved by shareholders in the 2025 Equity Incentive Plan. The general treatment of such awards is described below.
Retirement Benefits
Pursuant to outstanding awards under the Amicus Amended and Restated 2007 Equity Incentive Plan, all participants in the plan, including each named executive officer, would generally be eligible for the retirement benefits set forth below, upon such participant’s termination of employment, if such participant meets the following criteria at the time of such termination:
●The participant must have at least 5 years of continuous service
●The participant must be at least 55 years of age
●The sum of the participant’s age and years of service with the Company must equal or exceed 67 years (collectively, the “Retirement Criteria”)
Notwithstanding the above, if the participant’s employment is terminated by the Company for cause, then the retirement benefits would be forfeited. Ms. Rosenberg is the only named executive officer who met the Retirement Criteria in 2025. If an individual meets the Retirement Criteria, their awards upon separation, absent a termination for cause, is as follows:
●Options: Any unvested Options held by such participant that would have become vested and exercisable prior to the second anniversary of the participant’s separation, based solely on the participant's continued service through such time, will become exercisable on the date of such separation and all vested Options (including those eligible to vest pursuant to the preceding clause) shall remain exercisable until the earlier of (i) the 4th anniversary of the date of such separation, and (ii) the original expiration date of the term of the Option; any options not exercised in such period shall be forfeited with no further compensation due to the participant.
●RSUs: Any unvested RSUs that would vest up to the second anniversary of the participant’s separation, shall have accelerated vesting, and the shares will be delivered to the participant upon separation; any unvested RSUs beyond the two-year date shall be forfeited with no further compensation due to the participant.
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●PRSUs: As permitted under the plan and present in the applicable PRSU award agreements, a prorated portion of the participant’s PRSUs (based on the participant’s period of service with the Company during the performance period) would remain eligible to vest and become delivered based upon satisfaction of the goals applicable to such PRSUs (collectively, the “Equity Retirement Benefits”).
Disability
The same Equity Retirement Benefits described above afforded upon a participant’s retirement after satisfying the Retirement Criteria would be offered to any participant upon a termination of employment due to such participant’s disability if the termination had occurred on December 31, 2025.
Death
•Options: Any unvested Options will become immediately vested and exercisable, and all vested Options (including those that vest pursuant to the preceding clause) shall remain exercisable until the earlier of (i) the 4th anniversary of the date of such separation, and (ii) the original expiration date of the term of the Option; any options not exercised in such period shall be forfeited with no further compensation due to the participant.
•RSUs: Any unvested RSUs will become immediately vested.
•PRSUs: To the extent a participant's death occurs during an open performance period, such outstanding PRSUs will be prorated based on the number of days the participant was in service with the Company during the full measurement period, and such prorated PRSUs will remain outstanding and be earned or forfeited based on actual performance.
Change in Control
Under the terms of our equity award agreements, any unvested RSUs will generally automatically vest upon a change in control while unvested options and PRSUs are generally eligible to vest if a qualifying termination occurs following a change in control. The tables below do not include Mr. Campbell and Mr. Harford's RSU accelerations described in the section titled "280G Mitigation Actions" above as such RSUs were not outstanding as of December 31, 2025.
Potential Payments upon Termination without Cause
For each named executive officer, the following table sets forth quantitative estimates of the benefits that would have accrued if such executive’s employment had been terminated without cause on December 31, 2025 other than in connection with a change of control. Amounts below reflect potential payments pursuant to the severance agreements for such named executive officers.
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Name and Principal Position | Salary Continuation ($) | Bonus ($)(1) | Benefit Continuation ($)(2) | Value of RSU & PRSU Vesting ($)(3)(4) | Value of Stock Option Vesting ($)(4) | Total ($) | ||||||||||||||
Bradley L. Campbell | 1,237,500(5) | 928,125 | 68,654(6) | 4,403,225 | 1,137,826 | 7,775,330 | ||||||||||||||
| President and Chief Executive Officer | ||||||||||||||||||||
Simon Harford | 525,300(7) | 236,385 | 46,275 | 1,317,317 | 180,590 | 2,305,868 | ||||||||||||||
| Chief Financial Officer | ||||||||||||||||||||
Ellen S. Rosenberg | 535,600(7) | 241,020 | 35,967 | 2,061,365 | 214,995 | 3,088,947 | ||||||||||||||
| Chief Legal Officer and Corporate Secretary | ||||||||||||||||||||
| David M. Clark | 497,948(7) | 224,077 | 15,124 | 1,526,261 | 167,859 | 2,431,268 | ||||||||||||||
| Chief People Officer | ||||||||||||||||||||
Jeffrey P. Castelli | 512,595(7) | 230,668 | 29,132 | 1,779,267 | 187,000 | 2,738,661 | ||||||||||||||
| Chief Development Officer | ||||||||||||||||||||
(1)Bonus component paid in lump sum.
(2)Other than with respect to Mr. Campbell, benefits to be continued consist of premiums paid by the Company for 12 months.
(3)The PRSUs reported in the table are based on an assumed satisfaction of the applicable performance goals based on the tracking of such goals as of December 31, 2025. The actual number of PRSUs delivered to a named executive officer would depend on the satisfaction of each performance goal at the end of the applicable performance period. PRSUs granted in 2023 are not included as they would have vested in the normal course as a result of the executive’s continued employment through the end of 2025.
(4)Value of the equity that would accelerate upon such event is calculated using the closing stock price of $14.24 on December 31, 2025. In the case of stock options, the amount shown reflects the option “spread” (i.e., the difference between the option exercise price and the fair market value of our common stock) as of December 31, 2025 of stock options that would become non-forfeitable in this case, to the extent that the fair market value exceeds the exercise price.
(5)Base salary paid in installments over an 18-month period following such termination of employment.
(6)Benefits to be continued consist of premiums paid by the Company for 18 months.
(7)Base salary paid in installments over a 12-month period following such termination of employment.
Potential Payments upon a Change of Control
The following table sets forth quantitative estimates of the benefits that would have accrued to each of our named executive officers in the event of a change in control on December 31, 2025 under their applicable award agreements, without regard as to whether such named executive officer’s employment was terminated in connection with such change in control.
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Name and Principal Position | Value of RSU Vesting ($)(1) | ||||
Bradley L. Campbell | 4,545,593 | ||||
| President and Chief Executive Officer | |||||
Simon Harford | 500,066 | ||||
Chief Financial Officer | |||||
Ellen S. Rosenberg | 3,090,123 | ||||
| Chief Legal Officer and Corporate Secretary | |||||
| David M. Clark | 2,133,736 | ||||
Chief People Officer | |||||
| Jeffrey P. Castelli | 2,509,487 | ||||
Chief Development Officer | |||||
(1)The market value is based on the closing stock price of $14.24 on December 31, 2025.
Potential Payments upon Termination Due to Change of Control
The following table sets forth quantitative estimates of the benefits that would have accrued to each of our named executive officers if his or her employment had been terminated due to a termination without cause or a resignation with good reason on December 31, 2025, assuming that such termination occurs within twelve months following a change of control. The amounts in the table below are based upon assumptions required under SEC rules and differ from those set forth in the Merger Proxy Statement (which are governed by different rules).
Name and Principal Position | Salary Continuation ($) | Bonus ($)(1) | Benefit Continuation ($)(2) | Value of RSU & PRSU Vesting ($)(3) | Value of Stock Option Vesting ($)(3) | Total ($) | ||||||||||||||
Bradley L. Campbell | 1,650,000(4) | 1,237,500 | 91,539(5) | 10,531,206 | 2,595,604 | 16,105,849 | ||||||||||||||
| President and Chief Executive Officer | ||||||||||||||||||||
Simon Harford | 787,950(6) | 236,385 | 69,413 | 2,500,288 | 854,612 | 4,448,648 | ||||||||||||||
| Chief Financial Officer | ||||||||||||||||||||
Ellen S. Rosenberg | 803,400(6) | 241,020 | 53,951 | 5,591,037 | 1,070,824 | 7,760,232 | ||||||||||||||
| Chief Legal Officer and Corporate Secretary | ||||||||||||||||||||
David M. Clark | 746,922(6) | 224,077 | 22,686 | 3,806,039 | 676,637 | 5,476,360 | ||||||||||||||
Chief People Officer | ||||||||||||||||||||
Jeffrey P. Castelli | 768,893(6) | 230,668 | 43,698 | 4,509,708 | 805,532 | 6,358,499 | ||||||||||||||
Chief Development Officer | ||||||||||||||||||||
(1)Bonus component paid in lump sum.
(2)Other than with respect to Mr. Campbell, benefits to be continued consist of premiums paid by the Company for 18 months.
(3)Value of the equity that would accelerate upon such event is calculated using the closing stock price of $14.24 on December 31, 2025. In the case of stock options, the amount shown reflects the option “spread” (i.e., the difference between the option exercise price and the fair market value of our common stock) as of December 31, 2025 of stock options that would become non-forfeitable in this case, to the extent that the fair market value exceeds the exercise price. PRSUs granted in 2023 are not included as they would have vested in the normal course as a result of the executive’s continued employment through the end of 2025.
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(4)Base salary paid in installments over a 24-month period following such termination of employment.
(5)Benefits to be continued consist of premiums paid by the Company for 24 months.
(6)Base salary paid in installments over an 18-month period following such termination of employment.
Potential Payments upon Termination Due to Disability
The following sets forth quantitative estimates of the benefits that would have accrued to each of our named executive officers if his or her employment had been terminated due to disability on December 31, 2025.
Name and Principal Position | Benefit Continuation ($)(1) | Value of RSU & PRSU Vesting ($)(2) | Value of Stock Option Vesting ($)(2) | Total ($) | ||||||||||
Bradley L. Campbell | 45,770 | 6,994,756 | 1,429,381 | 8,469,907 | ||||||||||
| President and Chief Executive Officer | ||||||||||||||
Simon Harford | 46,275 | 1,521,633 | 516,623 | 2,084,531 | ||||||||||
Chief Financial Officer | ||||||||||||||
Ellen S. Rosenberg | 35,967 | 2,963,387 | 584,579 | 3,583,933 | ||||||||||
| Chief Legal Officer and Corporate Secretary | ||||||||||||||
David M. Clark | 15,124 | 2,155,701 | 373,723 | 2,544,548 | ||||||||||
Chief People Officer | ||||||||||||||
| Jeffrey P. Castelli | 29,132 | 2,530,954 | 446,879 | 3,006,965 | ||||||||||
Chief Development Officer | ||||||||||||||
(1)Benefits to be continued consist of benefit continuation and HSA premiums paid by the Company for 12 months following such termination.
(2)Value of the equity that would accelerate upon such event is calculated using the closing stock price of $14.24 on December 31, 2025. In the case of stock options, the amount shown reflects the option “spread” (i.e., the difference between the option exercise price and the fair market value of our common stock) as of December 31, 2025 of stock options that would become non-forfeitable in this case, to the extent that the fair market value exceeds the exercise price. PRSUs granted in 2023 are not included as they would have vested in the normal course as a result of the executive’s continued employment through the end of 2025.
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Potential Payments upon Termination Due to Death
The following sets forth quantitative estimates of the benefits that would have accrued to each of our named executive officers if his or her employment had been terminated due to death on December 31, 2025.
Name and Principal Position | Benefit Continuation ($)(1) | Value of RSU & PRSU Vesting ($)(2) | Value of Stock Option Vesting ($)(2) | Total ($) | ||||||||||
Bradley L. Campbell | 45,770 | 7,943,642 | 2,595,604 | 10,585,016 | ||||||||||
| President and Chief Executive Officer | ||||||||||||||
Simon Harford | 46,275 | 1,317,328 | 854,612 | 2,218,215 | ||||||||||
Chief Financial Officer | ||||||||||||||
Ellen S. Rosenberg | 35,967 | 3,989,108 | 1,070,824 | 5,095,899 | ||||||||||
| Chief Legal Officer and Corporate Secretary | ||||||||||||||
David M. Clark | 15,124 | 2,820,232 | 676,637 | 3,511,993 | ||||||||||
Chief People Officer | ||||||||||||||
| Jeffrey P. Castelli | 29,132 | 3,326,749 | 805,532 | 4,161,413 | ||||||||||
Chief Development Officer | ||||||||||||||
(1)Benefits to be continued consist of benefit continuation and HSA premiums paid by the Company for 12 months following such termination.
(2)Value of the equity that would accelerate upon such event is calculated using the closing stock price of $14.24 on December 31, 2025. In the case of stock options, the amount shown reflects the option “spread” (i.e., the difference between the option exercise price and the fair market value of our common stock) as of December 31, 2025 of stock options that would become non-forfeitable in this case, to the extent that the fair market value exceeds the exercise price. PRSUs granted in 2023 are not included as they would have vested in the normal course as a result of the executive’s continued employment through the end of 2025.
Potential Payments Due to Retirement
The following sets forth quantitative estimates of the benefits that would have accrued to Ms. Rosenberg on December 31, 2025 if she had elected to retire. Ms. Rosenberg was the only named executive officers to qualify for the retirement benefit as of December 31, 2025.
Name and Principal Position | Value of RSU & PRSU Vesting ($)(1) | Value of Stock Option Vesting ($)(1) | Total ($) | ||||||||
Ellen Rosenberg Chief Legal Officer and Corporate Secretary | 2,963,387 | 584,579 | 3,547,966 | ||||||||
(1)Value of the equity that would accelerate upon such event is calculated using the closing stock price of $14.24 on December 31, 2025. In the case of stock options, the amount shown reflects the option “spread” (i.e., the difference between the option exercise price and the fair market value of our common stock) as of December 31, 2025 of stock options that would become non-forfeitable in this case, to the extent that the fair market value exceeds the exercise price. PRSUs granted in 2023 are not included as they would have vested in the normal course as a result of the executive’s continued employment through the end of 2025.
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CEO Pay Ratio
Under Section 953(b) of the Dodd-Frank Wall Street Reform and Consumer Protection Act and Item 402(u) of Regulation S-K, we are required to provide the following disclosure regarding the ratio of the annual total compensation of our Chief Executive Officer to the annual total compensation of our median employee (the “Pay Ratio”).
We are utilizing the median employee selected in 2024 again in 2025 in accordance with SEC rules, as there has been no change in our employee population or employee compensation arrangements that we reasonably believe would result in a significant change to our pay ratio disclosure. In 2024 we identified our median employee by determining the 2024 total annualized target cash compensation for all of our global employees (excluding Mr. Campbell who was serving as the Chief Executive Officer at the time of the identification), whether employed on a full-time, part-time, or seasonal basis, who were employed by us on October 1, 2024 as our consistently applied compensation measure. Total annualized target cash compensation for these purposes consists of annualized base salary and annual target bonus. For 2025, we calculated such employee’s annual total compensation in the same manner that we determined the total compensation of our Chief Executive Officer for purposes of the Summary Compensation Table disclosed above.
For the year ended December 31, 2025, (i) the annual total compensation of our Chief Executive Officer was $8,946,931; (ii) the annual total compensation of our median employee was $283,420; and (iii) based on this information, we reasonably estimate our Pay Ratio to be 32:1. Because the SEC rules for identifying the median employee and calculating the Pay Ratio allow companies to use different methodologies, to apply certain exemptions, and to make reasonable estimates and assumptions, the Pay Ratio calculation presented above is a reasonable estimate and may not be comparable to the pay ratio reported by other companies.
Director Compensation
Pursuant to our Director Compensation Policy, each non-employee member of our Board received the following cash compensation for Board services during 2025, as applicable:
●$50,000 per year for service as a Board member;
●$75,000 per year for service as Chairman of the Board;
●$30,000 per year for service as chairperson of the Audit and Compliance Committee (inclusive of committee membership fees described below);
●$20,000 per year for service as chairperson of the Compensation and Leadership Development Committee (inclusive of committee membership fees described below);
●$12,500 per year for service as chairperson of the Nominating and Corporate Governance Committee (inclusive of committee membership fees described below);
●$16,000 per year for service as chairperson of the Science and Technology Committee for service in 2025 prior to July 1, 2025 and $17,000 per year for service as chairperson of the Science and Technology Committee, effective July 1, 2025 (inclusive of committee membership fees described below);
●$12,000 per year for service as a member of the Audit and Compliance Committee;
●$10,000 per year for service as a member of the Compensation and Leadership Development Committee;
●$7,500 per year for service as a member of the Nominating and Corporate Governance Committee; and
●$7,500 per year for service as a member of the Science and Technology Committee for service in 2025 prior to July 1, 2025 and $9,000 per year for service as a member of the Science and Technology Committee, effective July 1, 2025.
In 2025 each director received an annual grant of non-qualified options and RSUs with an approved value of $365,000, with 2/3 of the value assigned to non-qualified stock options, and 1/3 of the value assigned to RSUs. In determining the value of
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these grants, the Black-Scholes option pricing model is used to estimate the grant date fair value of stock options, whereas the value of RSUs is the fair market value of the shares of Common Stock underlying such RSUs on the date of grant. The grant date is the date of our Annual Meeting of Stockholders and each grant will vest in full on the first anniversary of the grant date. The exercise price of each option granted to a non-employee director will be equal to 100% of the fair market value of a share on the date of grant. Options will have a maximum term of 10 years measured from the grant date, subject to earlier termination in the event of the director’s cessation of Board service.
Newly appointed independent Board members receive non-qualified options and RSUs with an approved value of $500,000, consisting of 50% RSUs and 50% options. The number of RSUs and options subject to the grant is calculated in the same manner as set forth above with respect to our non-employee director annual grants. The exercise price of each option granted will be equal to 100% of the fair market value of a share on the date of the grant. Unlike the annual grant to our directors, but consistent with our grants to our named executive officers, these initial grant awards vest over a four-year period. The RSUs granted to each newly appointed director vest 25% per year on the anniversary of the grant date whereas the options will vest 25% on the first anniversary of the date of grant with the remainder vesting ratably each month thereafter over a three-year period. Vesting of these initial grant awards is conditioned on the director’s continued service through each vesting date.
In connection with the Transaction, outstanding options and unvested RSUs held by our directors will receive the same treatment as our executive officers as described under the heading "Transaction Related Treatment" above.
Director Compensation Table
The following table provides information regarding the compensation that each of our directors, with the exception of Mr. Campbell, our CEO, who appears in the named executive officer tables above, earned during the year ended December 31, 2025. Mr. Campbell does not receive any additional compensation in respect of his services as a director.
Name | Fees Earned or Paid in Cash ($)(1) | Stock Awards ($)(2) | Option Awards ($)(2) | All Other Compensation ($) | Total ($) | ||||||||||||
Lynn D. Bleil | 72,500 | 121,667 | 243,333 | — | 437,500 | ||||||||||||
Michael A. Kelly | 70,250 | 121,667 | 243,333 | — | 435,250 | ||||||||||||
Margaret G. McGlynn | 77,500 | 121,667 | 243,333 | — | 442,500 | ||||||||||||
Michael G. Raab | 132,500 | 121,667 | 243,333 | — | 497,500 | ||||||||||||
Eiry W. Roberts, M.D. | 68,250 | 121,667 | 243,333 | — | 433,250 | ||||||||||||
Glenn P. Sblendorio(3) | 90,000 | 121,667 | 243,333 | — | 455,000 | ||||||||||||
Craig A. Wheeler | 78,500 | 121,667 | 243,333 | — | 443,500 | ||||||||||||
Burke W. Whitman | 69,500 | 121,667 | 243,333 | — | 434,500 | ||||||||||||
(1)Represents fees earned by non-employee directors pursuant to the Compensation and Leadership Development Committee’s recommendation and Board approval.
(2)Represents the aggregate grant date fair value computed in accordance with FASB ASC Topic 718, Compensation—Stock Compensation. Assumptions made in this valuation are discussed in this Form 10-K at "— Note 9. Stock-based Compensation". Subject generally to continued service, annual non-employee director grants vest at the following year’s Annual Meeting while the initial non-employee director grants vest over four years.
(3)As of February 18, 2026, Mr. Sblendorio also has a cash balance of $78,913 in the Cash Deferral Plan that will be paid out in connection with, and subject to, the closing of the Transaction.
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As of December 31, 2025, our directors, with the exception of Mr. Campbell who appears in the named executive officer tables, had the following number of stock options outstanding:
Name | Aggregate Options Outstanding | Vested/Unvested | ||||||
Lynn D. Bleil | 281,999 | 207,127/74,872 | ||||||
Michael A. Kelly | 243,569 | 168,697/74,872 | ||||||
Margaret G. McGlynn | 312,542 | 237,670/74,872 | ||||||
Michael G. Raab | 312,542 | 237,670/74,872 | ||||||
Eiry W. Roberts, M.D. | 233,861 | 158,989/74,872 | ||||||
Glenn P. Sblendorio | 312,542 | 237,670/74,872 | ||||||
Craig A. Wheeler | 322,542 | 247,670/74,872 | ||||||
Burke W. Whitman | 273,885 | 199,013/74,872 | ||||||
Directors are also eligible to defer board fees pursuant to the terms of the Cash Deferral Plan and restricted stock units pursuant to the terms of the Stock Deferral Plan, each plan described more fully above.
As of December 31, 2025, our directors, with the exception of Mr. Campbell who appears in the named executive officer tables, had the following number of restricted stock units outstanding:
Name | Aggregate Restricted Stock Units Outstanding | ||||
Lynn D. Bleil | 75,736(1) | ||||
Michael A. Kelly | 20,414 | ||||
Margaret G. McGlynn | 63,631(2) | ||||
Michael G. Raab | 20,414 | ||||
Eiry W. Roberts, M.D. | 20,414 | ||||
Glenn P. Sblendorio | 20,414 | ||||
Craig A. Wheeler | 20,414 | ||||
Burke W. Whitman | 20,414 | ||||
(1)The number of restricted stock units outstanding includes 6,250 RSUs that vested on June 4, 2020, 5,855 RSUs that vested on June 4, 2021, 10,115 RSUs that vested on June 10, 2022, 12,671 RSUs that vested on June 9, 2023, 8,584 RSUs that vested on June 8, 2024, and 11,847 RSUs that vested on June 6, 2025 but were otherwise deferred or re-deferred to June 4, 2030, end of service, June 10, 2030, June 9, 2031, June 8, 2031, and June 10, 2027 respectively.
(2)The number of restricted stock units outstanding includes 10,115 RSUs that vested on June 10, 2022, 12,671 RSUs that vested on June 9, 2023, 8,584 RSUs that vested on June 8, 2024, and 11,847 RSUs that vested on June 6, 2025 but were otherwise deferred to June 10, 2026, June 9, 2027, June 8, 2028, and June 1, 2029 respectively.
Item 12. SECURITY OWNERSHIP OF CERTAIN BENEFICIAL OWNERS AND MANAGEMENT AND RELATED STOCKHOLDER MATTERS.
The following table sets forth certain information with respect to the beneficial ownership of our Common Stock as of February 12, 2026 for (a) the executive officers named in the Summary Compensation Table contained in this Annual Report on Form 10-K, (b) each of our directors, (c) all of our current directors and executive officers as a group and (d) each stockholder known by us to own beneficially more than 5% of our Common Stock. Beneficial ownership is determined in accordance with the rules of the SEC and includes voting or investment power with respect to the securities.
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We deem shares of Common Stock that may be acquired by an individual or group within 60 days of February 12, 2026 pursuant to the exercise of options or warrants to be outstanding for the purpose of computing the percentage ownership of such individual or group, but are not deemed to be outstanding for the purpose of computing the percentage ownership of any other person shown in the table. Except as indicated in footnotes to this table, we believe that the stockholders named in this table have sole voting and investment power with respect to all shares of Common Stock shown to be beneficially owned by them based on information provided to us by these stockholders. Percentage of ownership is based on 314,000,712 shares of Common Stock outstanding on February 12, 2026.
Unless otherwise indicated below, the address of each of the individuals named below is: c/o Amicus Therapeutics, Inc., 47 Hulfish Street, Princeton, New Jersey 08542.
Name and Address of Beneficial Owner | Number of Shares Beneficially Owned | Percentage Of Shares Beneficially Owned | ||||||
5% Stockholders | ||||||||
Entities affiliated with The Vanguard Group(1) | 30,871,374 | 9.8% | ||||||
100 Vanguard Blvd. | ||||||||
Malvern, PA 19355 | ||||||||
Entities affiliated with BlackRock, Inc.(2) | 25,848,965 | 8.2% | ||||||
55 East 52nd Street | ||||||||
New York, NY 10055 | ||||||||
Named Executive Officers and Directors | Number of Shares Beneficially Owned | Percentage Of Shares Beneficially Owned | ||||||
Bradley L. Campbell(3) | 2,320,862 | * | ||||||
Simon Harford(4) | 333,322 | * | ||||||
Ellen S. Rosenberg(5) | 991,925 | * | ||||||
David M. Clark(6) | 708,307 | * | ||||||
Jeffrey P. Castelli(7) | 907,738 | * | ||||||
Glenn P. Sblendorio(8) | 359,820 | * | ||||||
Michael G. Raab(9) | 333,113 | * | ||||||
Margaret G. McGlynn(10) | 268,670 | * | ||||||
Craig A. Wheeler(11) | 311,887 | * | ||||||
Lynn D. Bleil(12) | 245,332 | * | ||||||
Burke W. Whitman(13) | 297,197 | * | ||||||
Michael A. Kelly(14) | 220,751 | * | ||||||
Eiry W. Roberts, M.D.(15) | 214,850 | * | ||||||
All directors and executive officers as a group (13 persons) | 7,513,774 | 2.4% | ||||||
* Represents beneficial ownership of less than one percent of our outstanding Common Stock.
(1)This information is provided solely in reliance upon information included in a Form 13G/A filed with the SEC on December 3, 2025 by The Vanguard Group (“Vanguard”). As of November 28, 2025, Vanguard reported shared voting power of 2,120,104 shares of common stock, sole investment discretion of 28,380,963 shares of common stock and shared investment discretion of 2,490,411 shares of common stock.
(2)This information is provided solely in reliance upon information included in a Form 13F filed with the SEC on November 12, 2025 by BlackRock, Inc. (“BlackRock”). As of September 30, 2025, BlackRock reported sole
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voting power of 24,927,968 shares of common stock and sole dispositive power of 25,848,965 shares of common stock.
(3)Consists of 1,608,895 shares issuable upon the exercise of stock options exercisable within 60 days of February 12, 2026 and 711,967 shares held of record. Excludes shares issuable upon the exercise of stock options that are first exercisable after April 13, 2026 and unvested restricted stock units as of April 14, 2026.
(4)Consists of 224,844 shares issuable upon the exercise of stock options exercisable within 60 days of February 12, 2026 and 108,478 shares held of record. Excludes shares issuable upon the exercise of stock options that are first exercisable after April 13, 2026 and unvested restricted stock units as of April 14, 2026.
(5)Consists of 665,515 shares issuable upon the exercise of stock options exercisable within 60 days of February 12, 2026, 311,410 shares held directly by Ms. Rosenberg and 15,000 shares held by her spouse. Excludes shares issuable upon the exercise of stock options that are first exercisable after April 13, 2026 and both unvested and deferred restricted stock units as of April 14, 2026.
(6)Consists of 577,365 shares issuable upon the exercise of stock options exercisable within 60 days of February 12, 2026 and 130,942 shares held of record. Excludes shares issuable upon the exercise of stock options that are first exercisable after April 13, 2026 and unvested restricted stock units as of April 14, 2026.
(7)Consists of 589,321 shares issuable upon the exercise of stock options exercisable within 60 days of February 12, 2026 and 318,417 shares held of record. Excludes shares issuable upon the exercise of stock options that are first exercisable after April 13, 2026 and unvested restricted stock units as of April 14, 2026.
(8)Consists of 237,670 shares issuable upon the exercise of stock options exercisable within 60 days of February 12, 2026 and 122,150 shares held of record. Excludes shares issuable upon the exercise of stock options that are first exercisable after April 13, 2026 and unvested restricted stock units as of April 14, 2026.
(9)Consists of 237,670 shares issuable upon the exercise of stock options exercisable within 60 days of February 12, 2026 and 95,443 shares held of record. Excludes shares issuable upon the exercise of stock options that are first exercisable after April 13, 2026 and unvested restricted stock units as of April 14, 2026.
(10)Consists of 237,670 shares issuable upon the exercise of stock options exercisable within 60 days of February 12, 2026 and 31,000 shares held of record. Excludes shares issuable upon the exercise of stock options that are first exercisable after April 13, 2026 and both unvested and deferred restricted stock units as of April 14, 2026.
(11)Consists of 247,670 shares issuable upon the exercise of stock options exercisable within 60 days of February 12, 2026 and 64,217 shares held of record. Excludes shares issuable upon the exercise of stock options that are first exercisable after April 13, 2026 and unvested restricted stock units as of April 14, 2026.
(12)Consists of 207,127 shares issuable upon the exercise of stock options exercisable within 60 days of February 12, 2026 and 38,205 shares held of record. Excludes shares issuable upon the exercise of stock options that are first exercisable after April 13, 2026 and both unvested and deferred restricted stock units as of April 14, 2026.
(13)Consists of 199,013 shares issuable upon the exercise of stock options exercisable within 60 days of February 12, 2026 and 98,184 shares held of record. Excludes shares issuable upon the exercise of stock options that are first exercisable after April 13, 2026 and unvested restricted stock units as of April 14, 2026.
(14)Consists of 168,697 shares issuable upon the exercise of stock options exercisable within 60 days of February 12, 2026 and 52,054 shares held of record. Excludes shares issuable upon the exercise of stock options that are first exercisable after April 13, 2026 and unvested restricted stock units as of April 14, 2026.
(15)Consists of 158,989 shares issuable upon the exercise of stock options exercisable within 60 days of February 12, 2026 and 55,861 shares held of record. Excludes shares issuable upon the exercise of stock options that are first exercisable after April 13, 2026 and unvested restricted stock units as of April 14, 2026.
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POLICIES AND PROCEDURES FOR RELATED PARTY TRANSACTIONS
Item 13. CERTAIN RELATIONSHIPS AND RELATED TRANSACTIONS AND DIRECTOR INDEPENDENCE.
Certain Relationship and Related Transactions
Our Board maintains a formal written policy for the review of any transaction, arrangement or relationship in which Amicus is a participant and one of our executive officers, directors, director nominees, 5% stockholders (or their immediate family members), each of whom we refer to as a “related party,” has a direct or indirect interest. If a related party proposes to enter into such a transaction, arrangement, or relationship, which we refer to as a “related party transaction,” the related party must report the proposed transaction to our Chief Financial Officer. The proposed related party transaction must be reviewed and, if deemed appropriate, approved by the Board’s Audit and Compliance Committee prior to entry into such transaction, or ratified as soon as reasonably practicable after discovery that approval is required.
The Audit and Compliance Committee may approve or ratify the transaction only if the Audit and Compliance Committee determines that, under all of the circumstances, the transaction is not inconsistent with the Company’s best interests and does not violate its Code of Conduct and Ethics. Any related party transactions that are ongoing in nature will be reviewed annually. The Audit and Compliance Committee will review and consider such information regarding the related party transaction as it deems appropriate. In the last fiscal year, the Company did not enter into any transactions disclosable pursuant to Item 404(a) of Regulation S-K except that the Company paid $281,091 in membership dues to the Biotechnology Innovation Organization (“BIO”), a biotech trade association, of which Mr. Campbell is a director. The Company has paid a similar amount of membership dues to BIO in the past and expects to continue paying its annual dues in the future to retain its membership in BIO.
Our Board has reviewed the materiality of any relationship that each of our directors has with Amicus, either directly or indirectly, as well as other factors that may impact the independence determination for each of our directors. Based on this review, our Board has determined that the following directors are “independent directors” as defined by the rules and regulations of Nasdaq: Mses. Bleil and McGlynn, Messrs. Kelly, Raab, Sblendorio, Wheeler and Whitman, and Dr. Roberts.
Director Independence
Our Board has reviewed the materiality of any relationship that each of our directors has with Amicus, either directly or indirectly, as well as other factors that may impact the independence determination for each of our directors. Based on this review, our Board has determined that the following directors are “independent directors” as defined by the rules and regulations of Nasdaq: Mses. Bleil and McGlynn, Messrs. Kelly, Raab, Sblendorio, Wheeler and Whitman, and Dr. Roberts.
Item 14. PRINCIPAL ACCOUNTING FEES AND SERVICES.
Fees for Audit Services
The following table presents fees for professional audit services rendered by Ernst & Young LLP for the audit of our annual financial statements for the years ended December 31, 2025 and 2024, and fees billed for other services rendered by Ernst & Young LLP during those periods. All of such fees were approved by the Audit and Compliance Committee.
December 31, | ||||||||
2025 | 2024 | |||||||
Audit Fees | $ | 2,729,912 | $ | 2,555,948 | ||||
Audit-Related Fees | — | — | ||||||
Tax Fees | 20,600 | 20,600 | ||||||
All Other Fees | 232,166 | 28,183 | ||||||
Total | $ | 2,982,678 | $ | 2,604,731 | ||||
Fees for audit services included fees associated with the annual financial statement audit, an audit of our internal controls over financial reporting and reviews of the quarterly reports on Form 10-Q for both 2025 and 2024. In 2025 and 2024, the audit
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fees included costs of statutory audits of several of the Company’s foreign subsidiaries. The audit fees in 2025 and 2024 also include costs associated with comfort letters related to the ATM as well as filing of registration statements. Fees for tax services in 2025 and 2024 included fees associated with certain permitted compliance and advisory services. For 2025 all other fees included related to pricing compliance reporting for the U.K. and other advisory services. For 2024 all other fees included fees related to pricing compliance reporting for the U.K.
Policy on Audit and Compliance Committee Pre-Approval of Audit and Permissible Non-audit Services of Independent Registered Public Accounting Firm
Consistent with SEC policies regarding auditor independence, the Audit and Compliance Committee has responsibility for appointing, setting compensation and overseeing the work of the independent registered public accounting firm. In recognition of this responsibility, the Audit and Compliance Committee has established a policy to pre-approve all audit and permissible non-audit services provided by the independent registered public accounting firm.
Prior to engagement of the independent registered public accounting firm for the next year’s audit, management will submit an aggregate estimate of services expected to be rendered during that year for each of four categories of services to the Audit and Compliance Committee for approval.
1.Audit Services include audit work performed in the preparation of financial statements, as well as work that only the independent registered public accounting firm can reasonably be expected to provide, including comfort letters, statutory audits, and attest services and consultation regarding financial accounting and/or reporting standards.
2.Audit-Related Services are for assurance and related services that are traditionally performed by the independent registered public accounting firm, including due diligence related to mergers and acquisitions, employee benefit plan audits, and special procedures required to meet certain regulatory requirements.
3.Tax Services include all services performed by the independent registered public accounting firm’s tax personnel except those services specifically related to the audit of the financial statements, and includes fees in the areas of tax compliance, tax planning, and tax advice.
4.Other Fees are those associated with services not captured in the other categories.
Prior to engagement, the Audit and Compliance Committee pre-approves these services by category of service. The fees are budgeted, and the Audit and Compliance Committee requires the independent registered public accounting firm and management to report actual fees versus the budget periodically throughout the year by category of service. During the year, circumstances may arise when it may become necessary to engage the independent registered public accounting firm for additional services not contemplated in the original pre-approval. In those instances, the Audit and Compliance Committee requires specific pre-approval before engaging the independent registered public accounting firm.
The Audit and Compliance Committee may delegate pre-approval authority to one or more of its members. The member to whom such authority is delegated must report, for informational purposes only, any pre-approval decisions to the Audit and Compliance Committee at its next scheduled meeting.
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PART IV
Item 15. EXHIBITS, FINANCIAL STATEMENT SCHEDULE
1. Index to Consolidated Financial Statements
The following Consolidated Financial Statements are filed as part of this report:
Management's Report on Consolidated Financial Statements and Internal Control over Financial Reporting | 83 | ||||
Report of Independent Registered Public Accounting Firm (PCAOB ID: | 84 | ||||
Consolidated Balance Sheets as of December 31, 2025 and 2024 | 87 | ||||
Consolidated Statements of Operations for the years ended December 31, 2025, 2024 and 2023 | 88 | ||||
Consolidated Statements of Comprehensive Loss for the years ended December 31, 2025, 2024 and 2023 | 89 | ||||
Consolidated Statements of Changes in Stockholders' Equity for the years ended December 31, 2025, 2024 and 2023 | 90 | ||||
Consolidated Statements of Cash Flows for the years ended December 31, 2025, 2024 and 2023 | 91 | ||||
Notes To Consolidated Financial Statements | 93 | ||||
2. Consolidated Financial Statement Schedules
All schedules are omitted because they are not required or because the required information is included in the Consolidated Financial Statements or notes thereto.
3. Exhibits
| Incorporated by Reference to SEC Filing | ||||||||||||||||||||||||||||||||
| Exhibit No. | Filed Exhibit Description | Form | Date | Exhibit No. | Filed with this Form 10-K | |||||||||||||||||||||||||||
| 1.1 | Equity Distribution Agreement, dated November 7, 2022, by and between Amicus Therapeutics, Inc. and Goldman Sachs & Co. LLC | Form 8-K | 11/7/2022 | 1.1 | ||||||||||||||||||||||||||||
| 2.1 | Agreement and Plan of Merger, dated November 19, 2013, by and among Amicus Therapeutics, Inc., CB Acquisition Corp., Callidus BioPharma, Inc., and Cuong Do | Form 8-K | 2/12/2014 | 2.1 | ||||||||||||||||||||||||||||
| +2.2 | Agreement and Plan of Merger, dated July 5, 2016, by and among MiaMed, Inc., the Registrant and Minervas Merger Sub, Inc. | Form 8-K | 7/6/2016 | 2.1 | ||||||||||||||||||||||||||||
| +2.3 | Agreement and Plan of Merger, dated as of September 19, 2018, by and among Amicus Therapeutics, Inc., Columbus Merger Sub Corp., Celenex, Inc. and Shareholder Representative Services LLC, solely in its capacity as the Shareholders’ Representative | Form 8-K | 9/25/2018 | 2.1 | ||||||||||||||||||||||||||||
| ***2.4 | Agreement and Plan of Merger, dated as of December 19, 2025, by and among Amicus Therapeutics, Inc., BioMarin Pharmaceutical Inc., and Lynx Merger Sub 1, Inc. | Form 8-K | 12/19/2025 | 2.1 | ||||||||||||||||||||||||||||
| 3.1 | Restated Certificate of Incorporation of the Registrant. | Form 10-K | 2/28/2012 | 3.1 | ||||||||||||||||||||||||||||
| 3.2 | Second Amended and Restated By-laws of the Registrant. | Form 10-Q | 8/8/2023 | 3.4 | ||||||||||||||||||||||||||||
| 3.3 | Certificate of Amendment to the Registrant's Restated Certificate of Incorporation. | Form 8-K | 6/10/2015 | 3.1 | ||||||||||||||||||||||||||||
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| Incorporated by Reference to SEC Filing | ||||||||||||||||||||||||||||||||
| Exhibit No. | Filed Exhibit Description | Form | Date | Exhibit No. | Filed with this Form 10-K | |||||||||||||||||||||||||||
| 3.4 | Certificate of Amendment to the Restated Certificate of Incorporation | Form 8-K | 6/8/2018 | 3.1 | ||||||||||||||||||||||||||||
| 3.5 | Certificate of Amendment to the Restated Certificate of Incorporation | Form 8-K | 6/13/2023 | 3.1 | ||||||||||||||||||||||||||||
| 3.6 | Amendment to Amicus Therapeutics, Inc.’s Second Amended and Restated By-laws, dated December 19, 2025 | Form 8-K | 12/19/2025 | 3.1 | ||||||||||||||||||||||||||||
| 4.1 | Specimen Stock Certificate evidencing shares of common stock | S-1/A (333-141700) | 5/17/2007 | 4.1 | ||||||||||||||||||||||||||||
| 4.2 | Form of Indenture | Form S-3ASR | 2/19/2025 | 4.9 | ||||||||||||||||||||||||||||
| 4.3 | Description of the Registrant's securities | Form 10-K | 2/19/2025 | 4.3 | ||||||||||||||||||||||||||||
| 4.4 | Form of Pre-Funded Warrant | Form 8-K | 9/29/2021 | 4.1 | ||||||||||||||||||||||||||||
| 4.5 | Securities Purchase Agreement, dated September 29, 2021, by and between the Company and Redmile Group LLC | Form 8-K | 9/29/2021 | 10.3 | ||||||||||||||||||||||||||||
| 10.1 | Form of Director and Officer Indemnification Agreement | Form 8-K | 12/28/2022 | 10.1 | ||||||||||||||||||||||||||||
| *10.2 | Amended and Restated 2007 Director Option Plan and form of option agreement | Form 8-K | 6/18/2010 | 10.2 | ||||||||||||||||||||||||||||
| 10.3 | Securities Purchase Agreement, dated November 20, 2013 by and among the Company and the purchasers identified therein | Form 8-K | 11/20/2013 | 10.1 | ||||||||||||||||||||||||||||
| **10.4 | Second Restated Agreement, dated November 19, 2013 by and between the Registrant and Glaxo Group Limited | Form 10-K | 2/28/2024 | 10.4 | ||||||||||||||||||||||||||||
| *10.5 | Amicus Therapeutics, Inc. Amended and Restated Restricted Stock Unit Deferral Plan | Form 8-K | 12/28/2017 | 10.1 | ||||||||||||||||||||||||||||
| *10.6 | Amended and Restated 2007 Equity Incentive Plan | DEF 14A | 4/24/2024 | A | ||||||||||||||||||||||||||||
| *10.7 | Amicus Therapeutics, Inc. Cash Deferral Plan | Form 8-K | 10/28/2016 | 10.1 | ||||||||||||||||||||||||||||
| *10.8 | Amicus Therapeutics, Inc. 2025 Equity Incentive Plan | Form S-8 | 6/5/2025 | 99.2 | ||||||||||||||||||||||||||||
| *10.9 | Form of Performance-Based Restricted Stock Unit Award Agreement under the Amended and Restated 2007 Equity Incentive Plan | Form 8-K | 12/30/2016 | 10.1 | ||||||||||||||||||||||||||||
| *10.10 | Amendment #1 to the Amicus Therapeutics, Inc. Cash Deferral Plan. | Form 8-K | 10/26/2014 | 10.1 | ||||||||||||||||||||||||||||
| *10.11 | Amendment #2 to the Amicus Therapeutics, Inc. Cash Deferral Plan. | Form 8-K | 12/19/2019 | 10.1 | ||||||||||||||||||||||||||||
| *10.12 | Amendment #3 to the Amicus Therapeutics, Inc. Cash Deferral Plan | Form 10-Q | 11/9/2021 | 10.9 | ||||||||||||||||||||||||||||
| *10.13 | Employment Agreement, dated August 1, 2022, by and between the Registrant and Bradley L. Campbell. | Form 8-K | 8/1/2022 | 10.1 | ||||||||||||||||||||||||||||
| *10.14 | Employment Agreement dated February 18, 2020 between the Registrant and Ellen S. Rosenberg | Form 10-K | 3/2/2020 | 10.45 | ||||||||||||||||||||||||||||
| *10.15 | Employment Agreement dated February 18, 2020, between the Registrant and Daphne Quimi | Form 10-K | 3/2/2020 | 10.48 | ||||||||||||||||||||||||||||
| *10.16 | Employment Agreement, dated August 21, 2023, by and between Amicus Therapeutics, Inc. and Simon Harford | Form 10-Q | 11/8/2023 | 10.3 | ||||||||||||||||||||||||||||
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| Incorporated by Reference to SEC Filing | ||||||||||||||||||||||||||||||||
| Exhibit No. | Filed Exhibit Description | Form | Date | Exhibit No. | Filed with this Form 10-K | |||||||||||||||||||||||||||
| *10.17 | Employment Agreement dated February 18, 2020 between the Registrant and David Clark | Form 10-K | 3/2/2020 | 10.18 | ||||||||||||||||||||||||||||
| *10.18 | Employment Agreement dated February 18, 2020 between the Registrant and Jeffrey Castelli | Form 10-K | 3/2/2020 | 10.19 | ||||||||||||||||||||||||||||
| *10.19 | Form of Board Restricted Stock Unit Award Agreement under the Amended and Restated 2007 Equity Incentive Plan | Form 10-K | 3/1/2021 | 10.39 | ||||||||||||||||||||||||||||
| *10.20 | Form of Board Stock Option Award Agreement under the Amended and Restated 2007 Equity Incentive Plan | Form 10-K | 3/1/2021 | 10.41 | ||||||||||||||||||||||||||||
| *10.21 | Form of Stock Option Award Agreement under the Amended and Restated 2007 Equity Incentive Plan | Form 10-K | 3/1/2021 | 10.42 | ||||||||||||||||||||||||||||
| *10.22 | Form of Restricted Stock Unit Award Agreement under the Amended and Restated 2007 Equity Incentive Plan | Form 10-K | 2/24/2022 | 10.25 | ||||||||||||||||||||||||||||
| *10.23 | Form of Restricted Stock Unit Award Agreement under the Amicus Therapeutics, Inc. 2025 Equity Incentive Plan | Form 10-Q | 7/31/2025 | 10.5 | ||||||||||||||||||||||||||||
| *10.24 | Form of Restricted Stock Unit Award Agreement under the Amicus Therapeutics, Inc. 2025 Equity Incentive Plan | Form 10-Q | 7/31/2025 | 10.6 | ||||||||||||||||||||||||||||
| **10.25 | License Agreement dated December 22, 2022, by and between Amicus Therapeutics, Inc. and the Trustees of the University of Pennsylvania | Form 10-K | 3/1/2023 | 10.25 | ||||||||||||||||||||||||||||
| **10.26 | License Agreement, dated as of May 1, 2025, by and between Amicus Therapeutics, Inc. and Dimerix Biosciences Pty Ltd. | Form 10-Q | 7/31/2025 | 10.4 | ||||||||||||||||||||||||||||
| **10.27 | Amendment No. 1 to License Agreement, dated of as of October 20, 2025, by and between Amicus Therapeutics, Inc. and Dimerix Biosciences Pty Ltd. | X | ||||||||||||||||||||||||||||||
| **10.28 | Mutual Termination Agreement dated December 22, 2022, by and between Amicus Therapeutics, Inc. and the Trustees of the University of Pennsylvania. | Form 10-K | 3/1/2023 | 10.26 | ||||||||||||||||||||||||||||
| 10.29 | Loan Agreement, dated October 2, 2023 by and among Amicus Therapeutics, Inc., certain subsidiaries of Amicus Therapeutics, Inc. from time to time party thereto as Guarantors, Blackstone Alternative Credit Advisors LP, Blackstone Life Sciences Advisors L.L.C., certain lenders from time to time party thereto and Wilmington Trust, National Association, as Agent for the lenders. | Form 8-K | 10/2/2023 | 10.1 | ||||||||||||||||||||||||||||
| 10.30 | First Amendment to Loan Agreement, dated April 28, 2025 by and among Amicus Therapeutics, Inc., certain subsidiaries of Amicus Therapeutics, Inc. from time to time party thereto as Guarantors, Blackstone Alternative Credit Advisors LP, Blackstone Life Sciences Advisors L.L.C., certain lenders from time to time party thereto and Wilmington Trust, National Association, as Agent for the lenders. | Form 8-K | 5/1/2025 | 10.1 | ||||||||||||||||||||||||||||
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| Incorporated by Reference to SEC Filing | ||||||||||||||||||||||||||||||||
| Exhibit No. | Filed Exhibit Description | Form | Date | Exhibit No. | Filed with this Form 10-K | |||||||||||||||||||||||||||
| 10.31 | Second Amendment to Loan Agreement, dated June 16, 2025 by and among Amicus Therapeutics, Inc. certain subsidiaries of Amicus Therapeutics, Inc. from time to time party thereto as Guarantors, Blackstone Alternative Credit Advisors LP, Blackstone Life Sciences Advisors L.L.C., certain lenders from time to time party thereto and Wilmington Trust, National Association, as Agent for the lenders. | Form 10-Q | 7/31/2025 | 10.3 | ||||||||||||||||||||||||||||
| 10.32 | Securities Purchase Agreement, dated October 2, 2023, by and among Amicus Therapeutics, Inc. and the Purchasers identified on the signature pages thereto. | Form 8-K | 10/2/2023 | 10.2 | ||||||||||||||||||||||||||||
| **10.33 | Supply and Manufacturing Services Agreement, dated as of March 31, 2023, by and among the Company, WuXi Biologics (Hong Kong) Limited, WuXi Biologics Ireland Limited and WuXi Biologics Germany GmbH | Form 10-Q | 8/8/2023 | 10.2 | ||||||||||||||||||||||||||||
| **10.34 | First Amendment to the Supply and Manufacturing Services Agreement dated March 31, 2023 by and among the Company, WuXi Biologics (Hong Kong) Limited, WuXi Biologics Ireland Limited and WuXi Biologics Germany GmbH | Form 10-Q | 8/8/2024 | 10.1 | ||||||||||||||||||||||||||||
| **10.35 | Second Amendment to the Supply and Manufacturing Services Agreement dated March 31, 2023 by and among the Company, WuXi Biologics (Hong Kong) Limited, WuXi Biologics Ireland Limited and WuXi Biologics Germany GmbH | Form 10-Q | 11/4/2025 | 10.1 | ||||||||||||||||||||||||||||
| **10.36 | Third Amendment to the Supply and Manufacturing Services Agreement dated March 31, 2023 by and among the Company, WuXi Biologics (Hong Kong) Limited, WuXi Biologics Ireland Limited and WuXi Biologics Germany GmbH | Form 10-Q | 11/4/2025 | 10.2 | ||||||||||||||||||||||||||||
| 19.1 | Form 10-K | 2/19/2025 | 19.1 | |||||||||||||||||||||||||||||
| 21.1 | List of Subsidiaries | X | ||||||||||||||||||||||||||||||
| 23.1 | Consent of Independent Registered Public Accounting Firm. | X | ||||||||||||||||||||||||||||||
| 31.1 | Certification of Principal Executive Officer Pursuant to Rule 13a-14(a) of the Securities Exchange Act of 1934. | X | ||||||||||||||||||||||||||||||
| 31.2 | Certification of Principal Financial Officer Pursuant to Rule 13a-14(a) of the Securities Exchange Act of 1934. | X | ||||||||||||||||||||||||||||||
| 32.1 | Certificate of Principal Executive Officer pursuant to 18 U.S.C. Section 1350 and Section 906 of the Sarbanes-Oxley Act of 2002. | X | ||||||||||||||||||||||||||||||
| 32.2 | Certificate of Principal Financial Officer pursuant to 18 U.S.C. Section 1350 and Section 906 of the Sarbanes-Oxley Act of 2002. | X | ||||||||||||||||||||||||||||||
| 97.1 | Amicus Therapeutics, Inc. Clawback Policy | Form 10-K | 2/28/2024 | 97.1 | ||||||||||||||||||||||||||||
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| Incorporated by Reference to SEC Filing | ||||||||||||||||||||||||||||||||
| Exhibit No. | Filed Exhibit Description | Form | Date | Exhibit No. | Filed with this Form 10-K | |||||||||||||||||||||||||||
| 101 | The following financial information from this Annual Report on Form 10-K for the year ended December 31, 2025, formatted in Inline XBRL (Extensible Business Reporting Language) and filed electronically herewith: (i) the Consolidated Balance Sheets; (ii) the Consolidated Statements of Operations; (iii) the Consolidated Statements of Comprehensive Loss; (iv) the Consolidated Statements of Cash Flows; (v) and the Notes to the Consolidated Financial Statements. | X | ||||||||||||||||||||||||||||||
| 104 | The cover page from the Annual Report on Form 10-K for the year ended December 31, 2025 formatted in Inline XBRL (included in Exhibit 101). | X | ||||||||||||||||||||||||||||||
____________________________________________________________________
+ Confidential treatment has been granted as to certain portions of the document, which portions have been omitted and filed separately with the Securities and Exchange Commission.
* Indicates management contract or compensatory plan.
** Portions of the exhibit have been omitted in accordance with 17 CFR § 229.601(b)(10)(iv).
*** Certain exhibits and schedules have been omitted pursuant to 17 CFR § 229.601(a)(5) of Regulation S-K. The Company hereby undertakes to furnish supplemental copies of any of the omitted schedules upon request by the SEC; provided, that the Company may request confidential treatment pursuant to Rule 24b-2 of the Exchange Act for any schedules so furnished.
The information required by this item is incorporated by reference from the Proxy Statement under the captions "Certain Relationships and Related Transactions," "Director Independence," "Committee Compensation and Meetings of the Board of Directors," and "Compensation Committee Interlock and Insider Participation."
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Item 16. FORM 10-K SUMMARY.
Registrants may voluntarily include a summary of information required by Form 10-K under this Item 16. The Company has elected not to include such summary information.
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SIGNATURES
Pursuant to the requirements of Section 13 or 15(d) of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned, thereunto duly authorized on February 20, 2026.
AMICUS THERAPEUTICS, INC. (Registrant) | |||||
By: | /s/ Bradley L. Campbell | ||||
Bradley L. Campbell Chief Executive Officer | |||||
Pursuant to the requirements of the Securities Exchange Act of 1934, this Report has been signed below by the following persons on behalf of the Registrant and in the capacities and on the dates indicated.
Signature | Title | Date | ||||||||||||
| /s/ Bradley L. Campbell | President and Chief Executive Officer (Principal Executive Officer) | February 20, 2026 | ||||||||||||
| (Bradley L. Campbell) | ||||||||||||||
| /s/ Simon Harford | Chief Financial Officer (Principal Financial Officer) | February 20, 2026 | ||||||||||||
| (Simon Harford) | ||||||||||||||
| /s/ Samantha L. Prout | Chief Accounting Officer and Controller (Principal Accounting Officer) | February 20, 2026 | ||||||||||||
| (Samantha L. Prout) | ||||||||||||||
| /s/ Margaret G. McGlynn | Director | February 20, 2026 | ||||||||||||
| (Margaret G. McGlynn) | ||||||||||||||
| /s/ Michael G. Raab | Director | February 20, 2026 | ||||||||||||
| (Michael G. Raab) | ||||||||||||||
| /s/ Glenn Sblendorio | Director | February 20, 2026 | ||||||||||||
| (Glenn Sblendorio) | ||||||||||||||
| /s/ Craig Wheeler | Director | February 20, 2026 | ||||||||||||
| (Craig Wheeler) | ||||||||||||||
| /s/ Lynn Bleil | Director | February 20, 2026 | ||||||||||||
| (Lynn Bleil) | ||||||||||||||
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Signature | Title | Date | ||||||||||||
| /s/ Burke Whitman | Director | February 20, 2026 | ||||||||||||
| (Burke Whitman) | ||||||||||||||
| /s/ Michael A. Kelly | Director | February 20, 2026 | ||||||||||||
| (Michael A. Kelly) | ||||||||||||||
| /s/ Eiry W. Roberts, M.D. | Director | February 20, 2026 | ||||||||||||
| (Eiry W. Roberts, M.D.) | ||||||||||||||
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FAQ
What major strategic transaction does Amicus Therapeutics (FOLD) describe in its latest 10-K?
Amicus discloses a definitive merger agreement with BioMarin Pharmaceutical. BioMarin’s acquisition subsidiary will merge into Amicus, and each Amicus share will be converted into the right to receive $14.50 in cash, pending shareholder approval, regulatory clearances, and customary closing conditions.
How did Galafold perform financially for Amicus Therapeutics (FOLD) in 2025?
Galafold delivered $521.7 million of consolidated revenue in 2025. This was an increase of $63.6 million versus the prior year, reflecting continued commercial expansion in Fabry disease across more than 40 approved countries and broader geographic uptake of this oral precision therapy.
What were 2025 revenues for Pombiliti + Opfolda at Amicus Therapeutics (FOLD)?
Pombiliti + Opfolda generated $112.5 million in 2025 consolidated revenue. This represented a $42.3 million year-over-year increase, supported by regulatory approvals in the U.S., E.U., U.K., Canada, Australia, Switzerland, and Japan and growing reimbursement agreements across multiple countries.
What is Amicus Therapeutics’ (FOLD) cash position as of year-end 2025?
Amicus reports total cash, cash equivalents, and marketable securities of $293.5 million as of December 31 2025. This liquidity base is intended to support ongoing commercialization of Galafold and Pombiliti + Opfolda and further development of pipeline assets including licensed kidney candidate DMX‑200.
What rights did Amicus Therapeutics (FOLD) obtain for kidney drug candidate DMX-200?
Amicus signed an exclusive license with Dimerix for U.S. commercialization rights to Phase 3 FSGS candidate DMX‑200. Amicus paid a $30 million upfront fee and agreed to potential development, regulatory, and commercial milestones plus tiered royalties on U.S. net sales of DMX‑200.
What are key business risks highlighted by Amicus Therapeutics (FOLD) in this filing?
Amicus cites multiple risks including potential failure to close the BioMarin merger, heavy dependence on Galafold and Pombiliti + Opfolda, intense competition, regulatory and reimbursement pressures, manufacturing and supply reliance on third parties, substantial debt, and the possibility it may never achieve sustained profitability.
How many Amicus Therapeutics (FOLD) shares are outstanding and what is the non-affiliate market value?
The company reports 314,000,712 common shares outstanding as of February 12 2026. As of June 30 2025, the aggregate market value of 304,479,825 shares held by non-affiliates was approximately $1.74 billion, based on the NASDAQ Global Market closing price.
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