BioMarin cuts 2026 non-GAAP EPS outlook to $2.80–$3.10
Revenue guidance cites order timing uncertainty, while EPS guidance reflects charges tied to the Alesta acquisition and BMN 450 license.
Sentiment and the balance of points
Rhea-AI Sentiment reads the wording of the document, how positive or negative its language is on a 1 to 5 scale. The balance of points shown with the takes weighs what the document actually discloses, so the two can disagree, for example when a trial that missed its main goal is described in upbeat language.
Form Type
8-K
Rhea-AI Filing Summary
BioMarin Pharmaceutical Inc. (BMRN) reported third-quarter 2026 revenue of $1.025 billion, up 32% year over year. Metabolic Conditions revenue rose 44% to $744 million, while VOXZOGO revenue rose 12% to $245 million. GALAFOLD and POMBILITI + OPFOLDA contributed $152 million and $49 million, respectively, in their first full quarter as part of BioMarin. GAAP net loss was $123 million, versus $31 million a year earlier; Non-GAAP income was $45 million, versus $22 million. For the first nine months of 2026, operating cash flow was $577 million, versus $728 million in 2025.
BioMarin lowered 2026 Total Revenues guidance to $3.8 billion–$3.9 billion from $3.875 billion–$3.925 billion, and Non-GAAP diluted EPS guidance to $2.80–$3.10 from $4.90–$5.10. Revenue guidance reflects timing uncertainty for multiple orders from a single international customer. EPS guidance reflects anticipated charges and ongoing expenses associated with the Alesta acquisition and BMN 450 license agreement.
2 points · 0 major1 major · 4 points
How this balance works
Rhea-AI gives every point it takes from this document a weight. Minor counts 1, Moderate 3 and Major 9, so one Major point outweighs several Minor ones. The bar adds up the weights on each side, and when neither side holds more than 65% of the total the balance reads Mixed.
It reads the document as published, with the same rules for every company, and it does not look at what the market expected or at how the stock traded, so a point can be objectively good on a day the stock falls.
Rhea-AI Sentiment measures something else, the tone of the wording.
Hollow bars mark forward-looking points. How the balance works
Positive
- Moderate pointThird-quarter revenue rose 32% to $1.025 billion year over year.
- Moderate pointNon-GAAP income rose 105% to $45 million year over year.
Negative
- Major point. Forward-looking: it has not happened yet and may not happen.2026 revenue guidance fell to $3.8 billion–$3.9 billion from $3.875 billion–$3.925 billion.
- Moderate pointGAAP net loss widened to $123 million from $31 million year over year.
- Moderate pointNine-month operating cash flow fell to $577 million from $728 million.
- Minor point. Forward-looking: it has not happened yet and may not happen.2026 Non-GAAP diluted EPS guidance fell to $2.80–$3.10 from $4.90–$5.10.
Filing Explained
VOXZOGO’s hypochondroplasia application has a disclosed FDA target action date of May thirtieth, twenty twenty-seven, while the EMA has validated its submission.
BioMarin’s Form 8-K reports third-quarter 2026 results; at
The release attributes higher interest expense partly to debt issued to finance a portion of the Amicus acquisition.
For VOXZOGO in hypochondroplasia, the FDA set a target action date of
8-K Event Classification
2 items: 2.02, 9.01
2 items
Item 2.02
Results of Operations and Financial Condition
Financial
Disclosure of earnings results, typically an earnings press release or preliminary financials.
Item 9.01
Financial Statements and Exhibits
Exhibits
Financial statements, pro forma financial information, or exhibit attachments filed with this report.
Key Figures
Total Revenues: $1.025 billion
GAAP net loss: $123 million loss
Non-GAAP Income: $45 million
+5 more
8 metrics
Total Revenues
$1.025 billion
Third quarter of 2026; increased 32% year over year.
GAAP net loss
$123 million loss
Third quarter of 2026; compared with a $31 million loss in 2025.
Non-GAAP Income
$45 million
Third quarter of 2026; increased 105% year over year.
Metabolic Conditions revenue
$744 million
Third quarter of 2026; increased 44% year over year.
VOXZOGO revenue
$245 million
Third quarter of 2026; increased 12% year over year.
2026 Total Revenues guidance
$3.8 billion to $3.9 billion
Updated range; previous range was $3.875 billion to $3.925 billion.
2026 Non-GAAP diluted EPS guidance
$2.80 to $3.10 per share
Updated range; previous range was $4.90 to $5.10 per share.
Operating cash flow
$577 million
First nine months of 2026; compared with $728 million in the same period of 2025.
Key Terms
acquired in-process research and development (IPR&D), PDUFA target action date, Non-GAAP Diluted EPS, enzyme replacement therapy (ERT), +1 more
5 terms
acquired in-process research and development (IPR&D) financial
"acquired in-process research and development (IPR&D) charges"
PDUFA target action date regulatory
"PDUFA target action date of May 30, 2027"
The PDUFA target action date is the deadline set by the U.S. Food and Drug Administration (FDA) by which it aims to decide whether to approve or reject a new drug application. This date helps investors gauge when a company’s new medication might reach the market, potentially influencing sales and revenue expectations. It acts as a key milestone signaling progress in the drug approval process.
Non-GAAP Diluted EPS financial
"Non-GAAP Diluted EPS guidance"
Non-GAAP diluted EPS (Earnings Per Share) is a measure of a company's profit allocated to each share of stock, calculated using adjusted earnings that exclude certain items like one-time expenses or gains. It provides a view of ongoing performance by removing irregular or non-recurring factors. Investors use it to better understand the company's core profitability and compare performance across different periods or companies.
enzyme replacement therapy (ERT) medical
"investigational long-acting enzyme replacement therapy (ERT)"
Enzyme replacement therapy (ERT) is a medical treatment that supplies a missing or defective enzyme a patient’s body cannot make, restoring a chemical process much like adding the right tool to a broken machine so it can run again. For investors, ERT matters because successful therapies can create sustained revenue streams, affect regulatory risk and reimbursement decisions, and change the market value of companies developing, manufacturing, or distributing these specialized biologic drugs.
cost synergies financial
"approximately $280 million of GAAP cost synergies"
Cost synergies are the expected savings when two businesses combine activities so they can eliminate duplicate work, negotiate better prices, or run things more efficiently—like two households moving in together to share rent, groceries and utilities. Investors care because these savings can boost profit margins and cash flow, improving returns and supporting a higher valuation if the projected cuts are realistic and actually achieved. Actual results may differ from projections, so promised cost synergies are closely watched in deal assessments.
Earnings Snapshot
Total Revenues: $1.025 billion · Guidance included
Third quarter ended September 30, 2026
Total Revenues
$1.025 billion
32% year over year
GAAP net loss
$123 million loss
Compared with a $31 million loss in 2025
Non-GAAP Income
$45 million
105% year over year
Non-GAAP diluted EPS
$0.23 per share
92% year over year
Guidance
Updated 2026 guidance: Total Revenues of $3.8 billion to $3.9 billion and Non-GAAP diluted EPS of $2.80 to $3.10 per share.
FAQ
AI-generated questions and answers. How Rhea-AI works. Not financial advice.
How much revenue did BMRN report in the third quarter of 2026?
BioMarin reported third-quarter 2026 revenue of $1.025 billion, up 32% year over year. Metabolic Conditions revenue was $744 million, up 44%, and VOXZOGO revenue was $245 million, up 12%.
What is BioMarin's updated 2026 guidance?
BioMarin's updated 2026 guidance is $3.8 billion to $3.9 billion in Total Revenues and $2.80 to $3.10 in Non-GAAP diluted EPS. The previous ranges were $3.875 billion to $3.925 billion and $4.90 to $5.10, respectively.
What charges does BioMarin expect from Alesta and BMN 450?
For full-year 2026, BioMarin expects approximately $320 million in pre-tax charges for the Alesta acquisition and approximately $83 million for the BMN 450 license agreement. It estimates unfavorable impacts of approximately $1.55 and $0.32, respectively, to both GAAP and Non-GAAP diluted EPS.
When is the FDA action date for VOXZOGO in hypochondroplasia?
The FDA set a PDUFA target action date of May 30, 2027, for BioMarin's supplemental NDA to expand VOXZOGO use to hypochondroplasia. The European Medicines Agency also validated BioMarin's submission for approval to treat the condition.
What cost synergies does BioMarin expect from Amicus?
BioMarin continues to expect approximately $280 million in GAAP cost synergies and approximately $220 million on a Non-GAAP basis, to be fully realized in 2028.
AI-generated analysis. How Rhea-AI works. Not financial advice.
UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM 8-K
CURRENT REPORT
Pursuant to Section 13 OR 15(d) of The Securities Exchange Act of 1934
Date of Report (Date of earliest event reported): November 2, 2026
(Exact name of registrant as specified in its charter)
(State or other jurisdiction of incorporation or organization) | (Commission File Number) | (I.R.S. Employer Identification No.) | ||||||||||||
(Address of Principal Executive Offices) | (Zip Code) | |||||||||||||
(415 ) 506-6700
(Registrant's telephone number, including area code)
Not Applicable
(Former name or former address, if changed since last report.)
Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions (see General Instruction A.2. below):
Securities registered pursuant to Section 12(b) of the Act:
| Title of each class | Trading Symbol(s) | Name of each exchange on which registered | ||||||||||||
Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§240.12b-2 of this chapter).
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. ☐
o
Item 2.02 Results of Operations and Financial Condition.
On November 2, 2026, BioMarin Pharmaceutical Inc. (the Company) announced financial results for its third quarter ended September 30, 2026. The Company’s press release issued on November 2, 2026 is attached hereto as Exhibit 99.1 and is incorporated by reference herein.
The information in this Form 8-K, including in the press release furnished as Exhibit 99.1 hereto, shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the Exchange Act), or otherwise subject to the liabilities under that section or Sections 11 and 12(a)(2) of the Securities Act of 1933, as amended (the Securities Act), nor shall it be deemed incorporated by reference in any filing under the Securities Act or the Exchange Act, whether made before or after the date hereof, regardless of any general incorporation language in such filing.
Item 9.01 Financial Statements and Exhibits.
(d) Exhibits.
| Exhibit Number | Description | |||||||
99.1 | Press Release of the Company dated November 2, 2026 | |||||||
| 104 | Cover Page Interactive Data File - the cover page XBRL tags are embedded within the Inline XBRL document. | |||||||
SIGNATURES
Pursuant to the requirements 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.
| BioMarin Pharmaceutical Inc., a Delaware corporation | ||||||||
Date: November 2, 2026 | By: | /s/ G. Eric Davis | ||||||
| G. Eric Davis | ||||||||
| Executive Vice President, Chief Legal Officer | ||||||||
Exhibit 99.1
| Contact: | ||||||||
| Investors: | Media: | |||||||
| Traci McCarty | Marni Kottle | |||||||
| BioMarin Pharmaceutical Inc. | BioMarin Pharmaceutical Inc. | |||||||
| (415) 455-7558 | (650) 374-2803 | |||||||
BioMarin Reports Third Quarter 2026 Financial and Operating Results
Third Quarter 2026 Total Revenues Increased 32% Y/Y to $1.025 billion; Growth Driven by 44% Increase in Metabolic Conditions Y/Y, and 12% Increase in Skeletal Conditions Y/Y
Updates Full-year 2026 Guidance to Reflect Order Timing Uncertainty and Impact of Two Acquired In-process Research and Development (IPR&D) Charges
Recently Announced the Addition of a New Investigational Fabry Asset to Clinical Portfolio
PDUFA Action Date of May 30, 2027 for VOXZOGO® for the Treatment of Hypochondroplasia (HCH)
Conference Call and Webcast Scheduled Today at 4:30 p.m. ET
SAN RAFAEL, Calif., November 2, 2026 – BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced financial results for the third quarter ended September 30, 2026.
“BioMarin delivered a strong quarter, with revenue growth of 32%, driven by a more diversified portfolio following the acquisition of Amicus Therapeutics earlier this year and increased demand for our medicines from patients around the world. For the first time, quarterly revenue exceeded $1 billion, a milestone for BioMarin,” said Alexander Hardy, President and Chief Executive Officer of BioMarin. “We are building on this achievement by continuing to expand our pipeline through focused business development. We are excited to add our newest asset, an investigational treatment for Fabry disease, which has the potential to become the first subcutaneous enzyme therapy for this condition. With this fourth new asset added to our pipeline in 2026, we are following through on our commitment to build sustainable growth for the future.” Mr. Hardy added, “As we approach 2027, we are well positioned for our next chapter of growth, including the potential launch of VOXZOGO in hypochondroplasia and the continued execution of our financial and innovation strategy.”
1
2026 Business and Pipeline Highlights
Innovation
•BioMarin is accelerating innovation by expanding its pipeline through business development. Four transactions in 2026 have added development programs from preclinical through Phase 3:
◦BMN 450 (HM15421/GC1134A) is an investigational long-acting enzyme replacement therapy (ERT) that has the potential to be the first subcutaneous ERT for Fabry disease. Now in a global Phase 1/2 clinical study, it could extend BioMarin’s reach beyond patients with genetic variants amenable to GALAFOLD®.
◦BMN 440 (ALE1) for hypophosphatasia (HPP) is a potential first-in-class oral therapy for HPP. The Phase 1/2a study in healthy volunteers and adults with HPP is ongoing, with data expected in 2027.
◦BMN 430 for ReNU syndrome has the potential to be a first-in-disease antisense oligonucleotide (ASO) medicine for ReNU syndrome, a serious, rare neurodevelopmental condition with no approved targeted therapies. Preclinical studies are underway to identify a lead ASO candidate.
◦BMN 820 (DMX-200) for focal segmental glomerulosclerosis (FSGS) is a first-in-class oral CCR2 inhibitor for which BioMarin holds exclusive U.S. commercialization rights. The Phase 3 ACTION 3 trial is fully enrolled, with pivotal data expected in 2028.
•Today, BioMarin announced that the U.S. Food and Drug Administration (FDA) has set a PDUFA target action date of May 30, 2027 for its supplemental NDA to expand the use of VOXZOGO for the treatment of HCH, the first potential targeted therapy for the condition. BioMarin also announced today that the European Medicines Agency (EMA) validated its submission for the approval of VOXZOGO to treat HCH.
•BioMarin’s application for full approval of VOXZOGO in children with achondroplasia is under review by the FDA, with a PDUFA target action date of February 28, 2027. The application includes long-term safety and efficacy data, such as final adult height and clinical outcomes beyond linear growth.
•The Phase 2/3 study for BMN 333, BioMarin's long-acting C-type natriuretic peptide (CNP) for achondroplasia, continues to enroll on track. Dose-response data from this study is expected in 2027.
•In October, five-year results from the PROPEL open-label extension study with POMBILITI® + OPFOLDA® were presented in a late-breaking presentation at the World Muscle Society Annual Congress. In adults with late-onset Pompe disease, POMBILITI + OPFOLDA demonstrated durable motor function outcomes and relative stabilization of pulmonary function. This included people who had received enzyme replacement therapy for a mean of 7.6 years before enrolling. No new safety signals were identified.
•For BMN 351, BioMarin’s Phase 1/2 candidate for Duchenne muscular dystrophy, the company expects to provide a program update by year-end.
Growth
•In the first full quarter as part of BioMarin, GALAFOLD and POMBILITI + OPFOLDA delivered strong quarterly revenue, contributing $152 million and $49 million, respectively. The number of patients treated with GALAFOLD and POMBILITI + OPFOLDA increased by approximately 13% and 69% Y/Y, respectively. POMBILITI + OPFOLDA uptake benefited from supply constraints for other Pompe therapies.
•Metabolic Conditions revenue grew 44% Y/Y in the third quarter of 2026 and 26% Y/Y year-to-date. Growth was driven by the additions of GALAFOLD and POMBILITI + OPFOLDA and by higher VIMIZIM® revenue. The number of patients on therapy increased across all BioMarin-marketed Metabolic Conditions therapies, Y/Y.
•VOXZOGO revenue grew 12% Y/Y in the third quarter of 2026, and the number of children treated with VOXZOGO globally increased 17% Y/Y. As of the end of the quarter, children with achondroplasia in 58 countries around the world were being treated with VOXZOGO. More than 75% of VOXZOGO revenue was generated outside of the U.S., and a large international order previously expected in the third quarter is now anticipated in the fourth quarter. In the U.S., the majority of new patient starts were in children under the age of two.
2
Value Commitment
•BioMarin continued to integrate Amicus in the third quarter, realizing accelerated cost synergies to enable investments in initiatives to drive future growth of GALAFOLD and POMBILITI + OPFOLDA. BioMarin continues to expect approximately $280 million of GAAP cost synergies (approximately $220 million on a Non-GAAP basis) to be fully realized in 2028.
•On August 30, 2026, the company announced the global settlement of all outstanding claims with Ascendis Pharma A/S related to its product Yuviwel. As part of the agreement, Ascendis will pay BioMarin a royalty equal to 20% of net sales of Yuviwel in the U.S., retroactive to the first commercial sale, and 18% of net sales in the European Union, Brazil and South Korea until May 2030.
Third Quarter 2026 Financial Highlights
•Total Revenues for the third quarter of 2026 were $1,025 million, an increase of 32% compared to the same period in 2025, primarily driven by revenues from GALAFOLD and POMBILITI + OPFOLDA, which were acquired on April 27, 2026, as well as 18% revenue growth in VIMIZIM due to timing of large international orders. In the quarter, VOXZOGO revenue also increased by 12% due to new patients initiating therapy. These increases were partially offset by a decrease in NAGLAZYME® revenue due to timing of large international orders.
•GAAP Net Loss increased to $123 million compared to $31 million for the same period in 2025. The increase was primarily driven by the acquisition of Amicus Therapeutics (Amicus), including intangible asset amortization, interest expense from debt issued to finance a portion of the transaction, amortization of inventory fair value step-up, and integration and restructuring costs. The increase was also attributed to acquired in-process research and development (IPR&D) charges recorded in the quarter in connection with the acquisition of Alesta Therapeutics (Alesta). These increases were partially offset by revenue growth as described above.
•Non-GAAP Income for the third quarter of 2026 increased to $45 million compared to $22 million for the same period in 2025. The increase was primarily driven by revenue growth, partially offset by higher acquired IPR&D charges and higher interest expense, as described above. The increases were also partially offset by higher sales and marketing spend related to GALAFOLD and POMBILITI + OPFOLDA.
3
Financial Highlights (in millions of U.S. dollars, except percentages and per share data, unaudited)
| Three Months Ended September 30, | Nine Months Ended September 30, | ||||||||||||||||||||||||||||||||||
| 2026 | 2025 | % Change | 2026 | 2025 | % Change | ||||||||||||||||||||||||||||||
| Total Revenues | $1,025 | $776 | 32% | $2,781 | $2,347 | 18% | |||||||||||||||||||||||||||||
| Net Product Revenues by Product: | |||||||||||||||||||||||||||||||||||
| VOXZOGO | $245 | $218 | 12% | $717 | $654 | 10% | |||||||||||||||||||||||||||||
| Metabolic Conditions: | |||||||||||||||||||||||||||||||||||
| VIMIZIM | $216 | $183 | 18% | $620 | $587 | 6% | |||||||||||||||||||||||||||||
| NAGLAZYME | 108 | 122 | (11)% | 373 | 365 | 2% | |||||||||||||||||||||||||||||
PALYNZIQ® | 111 | 109 | 2% | 336 | 308 | 9% | |||||||||||||||||||||||||||||
| GALAFOLD | 152 | — | NM | 258 | — | NM | |||||||||||||||||||||||||||||
BRINEURA® | 53 | 48 | 10% | 151 | 137 | 10% | |||||||||||||||||||||||||||||
ALDURAZYME® | 57 | 54 | 6% | 137 | 159 | (14)% | |||||||||||||||||||||||||||||
| POMBILITI + OPFOLDA | 49 | — | NM | 79 | — | NM | |||||||||||||||||||||||||||||
| Total Metabolic Conditions Revenue | $744 | $516 | 44% | $1,954 | $1,556 | 26% | |||||||||||||||||||||||||||||
KUVAN® | $23 | $24 | (4)% | $71 | $76 | (7)% | |||||||||||||||||||||||||||||
ROCTAVIAN®(1) | $1 | $3 | (67)% | $15 | $23 | (35)% | |||||||||||||||||||||||||||||
| GAAP Net Income (Loss) | $(123) | $(31) | 297% | $27 | $395 | (93)% | |||||||||||||||||||||||||||||
Non-GAAP Income (2) | $45 | $22 | 105% | $430 | $525 | (18)% | |||||||||||||||||||||||||||||
GAAP Operating Margin % (3) | (8.0)% | (6.0)% | 5.7% | 19.4% | |||||||||||||||||||||||||||||||
Non-GAAP Operating Margin % (2) | 13.4% | 2.8% | 24.6% | 26.3% | |||||||||||||||||||||||||||||||
GAAP Diluted EPS | $(0.63) | $(0.16) | 294% | $0.14 | $2.04 | (93)% | |||||||||||||||||||||||||||||
Non-GAAP Diluted EPS (2) | $0.23 | $0.12 | 92% | $2.18 | $2.69 | (19)% | |||||||||||||||||||||||||||||
NM Percentage change is not meaningful for products acquired from Amicus on April 27, 2026.
(1) In 2026, the company announced that it will no longer market ROCTAVIAN.
(2) Refer to Non-GAAP Information beginning on page 10 of this press release for definitions of Non-GAAP Income, Non-GAAP Operating Margin percentage and Non-GAAP Diluted EPS along with the related reconciliations to the comparable information reported under U.S. GAAP.
(3) GAAP Operating Margin percentage is defined by the company as GAAP Income (Loss) from Operations divided by Total Revenues.
4
Forward-Looking Non-GAAP Financial Information
BioMarin does not provide guidance for GAAP reported financial measures (other than revenue) or a reconciliation of forward-looking Non-GAAP financial measures to the most directly comparable GAAP reported financial measures because the company is unable to predict with reasonable certainty the financial impact of changes resulting from its strategic portfolio and business operating model reviews; potential future asset impairments; gains and losses on investments; and other unusual gains and losses without unreasonable effort. These items are uncertain, depend on various factors, and could have a material impact on GAAP reported results for the guidance period. As such, any reconciliations provided would imply a degree of precision that could be confusing or misleading to investors.
Updated 2026 Full-Year Financial Guidance (in millions, except EPS amounts)
•Revenue: Total Revenues, Metabolic Conditions, and VOXZOGO guidance updated to reflect uncertainty in the timing of multiple orders from a single international customer.
•Non-GAAP Diluted EPS: BioMarin expects the Alesta acquisition and BMN 450 license agreement to result in total pre-tax charges of approximately $320 million and $83 million, respectively, for the full year 2026, consisting of the acquired IPR&D charges and ongoing operating expenses, representing an unfavorable impact of approximately $1.55 and $0.32, respectively, to both GAAP Diluted EPS and Non-GAAP Diluted EPS for the full year 2026.
| Item | Provided on August 6, 2026 | Updated November 2, 2026 | Midpoint Growth (Y/Y) | |||||||||||||||||||||||||||||||||||
| Total Revenues | $3,875 | to | $3,925 | $3,800 | to | $3,900 | 20% | |||||||||||||||||||||||||||||||
| Metabolic Conditions | $2,725 | to | $2,775 | $2,675 | to | $2,750 | 29% | |||||||||||||||||||||||||||||||
| VOXZOGO | $1,000 | to | $1,050 | $975 | to | $1,025 | 8% | |||||||||||||||||||||||||||||||
Other Revenues(1) | $100 | to | $125 | $125 | to | $150 | ||||||||||||||||||||||||||||||||
Non-GAAP Diluted EPS (2)(3) | $4.90 | to | $5.10 | $2.80 | to | $3.10 | (6)% | |||||||||||||||||||||||||||||||
(1) Other Revenues includes KUVAN, ROCTAVIAN, and royalties.
(2) Refer to Non-GAAP Information beginning on page 10 of this press release for definition of Non-GAAP Diluted EPS.
(3) Non-GAAP Diluted EPS guidance assumes approximately 200 million Weighted-Average Diluted Shares Outstanding.
BioMarin will host a conference call and webcast to discuss third quarter 2026 financial results today, Monday, November 2, 2026, at 4:30 p.m. ET. This event can be accessed through this link or on the investor section of the BioMarin website at www.biomarin.com.
| U.S./Canada Dial-in Number:+1 833-461-5787 | Replay Dial-in Number: +1 833-309-1852 | ||||
| International Dial-in Number: +1 626-884-3620 | Replay International Dial-in Number: +44 800 007 4044 | ||||
| Conference ID: 324 588 583 | Conference ID: 324 588 583 | ||||
About BioMarin
BioMarin is a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. Founded in 1997, the San Rafael, California-based company has a proven track record of innovation, with nine commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin seeks to unleash the full potential of genetic science by pursuing category-defining medicines that have a profound impact on patients. To learn more, please visit www.biomarin.com.
5
Forward-Looking Statements
This press release and the associated conference call and webcast contain forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: future financial performance, including the expectations of Revenues and Non-GAAP Diluted EPS for, in certain instances, the full-year 2026 and fourth quarter of 2026, and future periods, and the underlying drivers of those results, such as the expected demand and continued growth of BioMarin’s Metabolic Conditions portfolio, including PALYNZIQ, GALAFOLD and POMBILITI + OPFOLDA, and VOXZOGO, and the expected impact of the acquisition of Alesta Therapeutics B.V. (Alesta) and the BMN 450 license agreement; the anticipated benefits of the acquisition of Amicus Therapeutics, Inc., including the expected amount and timing of cost synergies as well as expected revenue from the addition of GALAFOLD and POMBILITI + OPFOLDA; the anticipated benefits of the acquisition of Alesta; the anticipated benefits of the BMN 450 license agreement; the anticipated benefits of the global settlement with Ascendis Pharma A/S, including BioMarin’s expected receipt of royalty payments on net sales of Yuviwel in the United States, the European Union, Brazil and South Korea; BioMarin’s plans for investment in innovation and future growth; the timing of orders for commercial products; plans and expectations regarding the development, commercialization and commercial prospects of BioMarin’s product candidates and commercial products, including the prospects and timing of actions relating to clinical studies and trials and product approvals, such as study initiations, study advancements, data readouts, submissions, filings, approvals, and label expansions; the expected benefits and availability of BioMarin’s commercial products and product candidates, including with respect to the potential new indication for VOXZOGO in hypochondroplasia; and potential growth opportunities and trends, including the assumptions and expectations regarding total addressable patient population (TAPP) with respect to the conditions targeted by BioMarin’s product candidates and commercial products.
These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: BioMarin’s success in the commercialization of its commercial products; BioMarin’s ability to successfully integrate and realize the anticipated benefits of any acquisitions and license agreements; BioMarin’s ability to accurately estimate future financial performance; impacts of macroeconomic and other external factors on BioMarin’s operations, regulatory uncertainty, the impact of new or increased tariffs, other trade protection measures, and escalating trade tensions; geopolitical instability, wars and military conflicts; results and timing of current and planned preclinical studies and clinical trials and the release of data from those trials; BioMarin’s ability to successfully manufacture its commercial products and product candidates; the content and timing of decisions by the U.S. Food and Drug Administration, the European Medicines Agency, the European Commission and other regulatory authorities concerning each of the described products and product candidates; the market for each of these products; BioMarin’s ability to meet product demand; actual sales of BioMarin’s commercial products; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption “Risk Factors” in BioMarin's Quarterly Report on Form 10-Q for the quarter ended June 30, 2026, as such factors may be updated by any subsequent reports. Investors are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.
BioMarin®, VOXZOGO®, VIMIZIM®, NAGLAZYME®, PALYNZIQ®, BRINEURA®, KUVAN®, ROCTAVIAN®, GALAFOLD®, and POMBILITI® + OPFOLDA® are registered trademarks of BioMarin Pharmaceutical Inc., or its affiliates. ALDURAZYME® is a registered trademark of BioMarin/Genzyme LLC. All other brand names and service marks, trademarks and other trade names appearing in this release are the property of their respective owners.
6
BIOMARIN PHARMACEUTICAL INC.
CONDENSED CONSOLIDATED STATEMENTS OF INCOME
Three and Nine Months Ended September 30, 2026 and 2025
(In thousands of U.S. dollars, except per share amounts)
(Unaudited)
| Three Months Ended September 30, | Nine Months Ended September 30, | ||||||||||||||||||||||
| 2026 | 2025 | 2026 | 2025 | ||||||||||||||||||||
| REVENUES: | |||||||||||||||||||||||
| Net product revenues | $ | 1,013,080 | $ | 760,812 | $ | 2,757,551 | $ | 2,308,438 | |||||||||||||||
| Royalty and other revenues | 11,566 | 15,321 | 23,011 | 38,250 | |||||||||||||||||||
| Total revenues | 1,024,646 | 776,133 | 2,780,562 | 2,346,688 | |||||||||||||||||||
| OPERATING EXPENSES: | |||||||||||||||||||||||
| Cost of sales | 205,416 | 140,085 | 603,210 | 441,733 | |||||||||||||||||||
| Research and development | 180,525 | 188,515 | 566,273 | 508,554 | |||||||||||||||||||
| Acquired in-process research and development (IPR&D) | 287,685 | 220,963 | 287,685 | 220,963 | |||||||||||||||||||
| Selling, general and administrative | 333,643 | 268,415 | 987,482 | 706,810 | |||||||||||||||||||
| Intangible asset amortization | 99,458 | 4,847 | 177,433 | 14,540 | |||||||||||||||||||
| Total operating expenses | 1,106,727 | 822,825 | 2,622,083 | 1,892,600 | |||||||||||||||||||
INCOME (LOSS) FROM OPERATIONS | (82,081) | (46,692) | 158,479 | 454,088 | |||||||||||||||||||
| Interest income | 4,630 | 17,854 | 37,670 | 55,694 | |||||||||||||||||||
| Interest expense | (55,628) | (2,579) | (133,881) | (8,121) | |||||||||||||||||||
| Other income, net | 1,310 | 5,093 | 8,550 | 7,972 | |||||||||||||||||||
INCOME (LOSS) BEFORE INCOME TAXES | (131,769) | (26,324) | 70,818 | 509,633 | |||||||||||||||||||
| Provision for (benefit from) income taxes | (8,938) | 4,420 | 43,360 | 114,159 | |||||||||||||||||||
NET INCOME (LOSS) | $ | (122,831) | $ | (30,744) | $ | 27,458 | $ | 395,474 | |||||||||||||||
EARNINGS (LOSS) PER SHARE, BASIC | $ | (0.63) | $ | (0.16) | $ | 0.14 | $ | 2.06 | |||||||||||||||
EARNINGS (LOSS) PER SHARE, DILUTED | $ | (0.63) | $ | (0.16) | $ | 0.14 | $ | 2.04 | |||||||||||||||
| Weighted average common shares outstanding, basic | 193,590 | 192,032 | 193,171 | 191,639 | |||||||||||||||||||
| Weighted average common shares outstanding, diluted | 193,590 | 192,032 | 195,214 | 196,893 | |||||||||||||||||||
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BIOMARIN PHARMACEUTICAL INC.
CONDENSED CONSOLIDATED BALANCE SHEETS
September 30, 2026 and December 31, 2025
(In thousands of U.S. dollars, except per share amounts)
| September 30, 2026 | December 31, 2025 | ||||||||||
| (Unaudited) | |||||||||||
| ASSETS | |||||||||||
| Current assets: | |||||||||||
| Cash and cash equivalents | $ | 736,007 | $ | 1,311,679 | |||||||
| Short-term investments | — | 248,930 | |||||||||
| Accounts receivable, net | 1,076,355 | 908,214 | |||||||||
| Inventory | 1,796,069 | 1,298,883 | |||||||||
| Other current assets | 312,205 | 185,784 | |||||||||
| Total current assets | 3,920,636 | 3,953,490 | |||||||||
| Noncurrent assets: | |||||||||||
| Long-term investments | — | 492,242 | |||||||||
| Property, plant and equipment, net | 980,081 | 952,508 | |||||||||
| Intangible assets, net | 4,777,247 | 213,837 | |||||||||
| Goodwill | 666,367 | 196,199 | |||||||||
| Deferred tax assets | 897,787 | 1,508,697 | |||||||||
| Other assets | 327,113 | 277,049 | |||||||||
| Total assets | $ | 11,569,231 | $ | 7,594,022 | |||||||
| LIABILITIES AND STOCKHOLDERS’ EQUITY | |||||||||||
| Current liabilities: | |||||||||||
| Accounts payable and accrued liabilities | $ | 946,107 | $ | 759,031 | |||||||
| Current portion of long-term debt, net | 658,717 | — | |||||||||
| Total current liabilities | 1,604,824 | 759,031 | |||||||||
| Noncurrent liabilities: | |||||||||||
| Long-term debt, net | 3,514,974 | 597,176 | |||||||||
| Other long-term liabilities | 193,401 | 150,816 | |||||||||
| Total liabilities | 5,313,199 | 1,507,023 | |||||||||
| Stockholders’ equity: | |||||||||||
Common stock, $0.001 par value: 500,000,000 shares authorized; 193,676,831 and 192,300,101 shares issued and outstanding, respectively | 194 | 192 | |||||||||
| Additional paid-in capital | 6,089,036 | 5,956,582 | |||||||||
| Company common stock held by the Nonqualified Deferred Compensation Plan | (10,851) | (10,508) | |||||||||
Accumulated other comprehensive loss | (4,011) | (13,473) | |||||||||
Retained earnings | 181,664 | 154,206 | |||||||||
| Total stockholders’ equity | 6,256,032 | 6,086,999 | |||||||||
| Total liabilities and stockholders’ equity | $ | 11,569,231 | $ | 7,594,022 | |||||||
8
BIOMARIN PHARMACEUTICAL INC.
CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS
Nine Months Ended September 30, 2026 and 2025
(In thousands of U.S. dollars)
(Unaudited)
| Nine Months Ended September 30, | |||||||||||
| 2026 | 2025 | ||||||||||
| CASH FLOWS FROM OPERATING ACTIVITIES: | |||||||||||
| Net income | $ | 27,458 | $ | 395,474 | |||||||
| Adjustments to reconcile net income to net cash provided by operating activities: | |||||||||||
| Depreciation and amortization | 244,514 | 59,119 | |||||||||
| Non-cash interest expense | 30,548 | 1,969 | |||||||||
| Stock-based compensation | 172,684 | 134,496 | |||||||||
| Impairment of assets | 15,203 | 2,967 | |||||||||
| Acquired IPR&D expense | 282,258 | 220,963 | |||||||||
| Deferred income taxes | (27,490) | 48,125 | |||||||||
| Unrealized foreign exchange losses (gains) | (8,380) | 244 | |||||||||
| Other | (7,091) | (5,399) | |||||||||
| Changes in operating assets and liabilities, net of effects of business acquired: | |||||||||||
| Accounts receivable, net | (60,435) | (107,378) | |||||||||
| Inventory | (1,621) | (98,796) | |||||||||
| Other current assets | (54,369) | (32,125) | |||||||||
| Other assets | 12,859 | (32,679) | |||||||||
| Accounts payable and accrued liabilities | (52,675) | 119,478 | |||||||||
| Other long-term liabilities | 3,511 | 21,892 | |||||||||
| Net cash provided by operating activities | 576,974 | 728,350 | |||||||||
| CASH FLOWS FROM INVESTING ACTIVITIES: | |||||||||||
| Purchases of property, plant and equipment | (78,032) | (62,316) | |||||||||
| Maturities and sales of investments | 767,277 | 294,547 | |||||||||
| Purchases of investments | (25,792) | (313,262) | |||||||||
| Business combination, net of cash acquired | (5,067,630) | — | |||||||||
Purchases of IPR&D, net of cash acquired | (277,881) | (285,193) | |||||||||
| Other investing activities, net | (1,191) | (5,569) | |||||||||
| Net cash used in investing activities | (4,683,249) | (371,793) | |||||||||
| CASH FLOWS FROM FINANCING ACTIVITIES: | |||||||||||
| Proceeds from exercises of awards under equity incentive plans | 6,655 | 7,714 | |||||||||
| Taxes paid related to net share settlement of equity awards | (43,330) | (53,265) | |||||||||
| Proceeds from borrowings | 3,650,000 | — | |||||||||
| Payments of debt issuance costs | (65,605) | — | |||||||||
| Repayments of borrowings | (15,000) | — | |||||||||
| Net cash provided by (used in) financing activities | 3,532,720 | (45,551) | |||||||||
| Effect of exchange rate changes on cash | (2,117) | (3,740) | |||||||||
| NET INCREASE (DECREASE) IN CASH AND CASH EQUIVALENTS | (575,672) | 307,266 | |||||||||
| Cash and cash equivalents: | |||||||||||
| Beginning of period | $ | 1,311,679 | $ | 942,842 | |||||||
| End of period | $ | 736,007 | $ | 1,250,108 | |||||||
9
Non-GAAP Information
The results presented in this press release include both GAAP information and Non-GAAP information. Non-GAAP Income is defined by the company as GAAP Net Income (Loss) excluding amortization, stock-based compensation expense and, in certain periods, certain other specified items, as detailed below when applicable. The company also includes a Non-GAAP adjustment for the estimated tax impact of the reconciling items. Non-GAAP Cost of Sales (COS), Non-GAAP R&D expenses and Non-GAAP Selling, General and Administrative (SG&A) expenses are defined by the company as GAAP COS, GAAP R&D expenses and GAAP SG&A expenses, respectively, excluding stock-based compensation expense and, in certain periods, certain other specified items, as detailed below when applicable. Non-GAAP Operating Margin percentage is defined by the company as GAAP Income (Loss) from Operations, excluding amortization of intangible assets, stock-based compensation expense and, in certain periods, certain other specified items, divided by GAAP Total Revenues. Non-GAAP Diluted EPS is defined by the company as Non-GAAP Income divided by Non-GAAP Weighted-Average Diluted Shares Outstanding. Non-GAAP Weighted-Average Diluted Shares Outstanding is defined by the company as GAAP Weighted-Average Diluted Shares Outstanding, adjusted to include any common shares issuable under the company’s equity plans or convertible debt in periods when they are dilutive under Non-GAAP. Non-GAAP synergies is defined by the company as Amicus’ legacy labor and external spend cost reductions, excluding the impact of stock-based compensation.
BioMarin regularly uses both GAAP and Non-GAAP results and expectations internally to assess its financial operating performance and evaluate key business decisions related to its principal business activities: the discovery, development, manufacture, marketing and sale of innovative biologic therapies. BioMarin also uses Non-GAAP Income internally to understand, manage and evaluate its business and to make operating decisions, and compensation of executives is based in part on this measure. Because these Non-GAAP metrics are important internal measurements for BioMarin, the company believes that providing this information in conjunction with BioMarin’s GAAP information enhances investors’ and analysts’ ability to meaningfully compare the company’s results from period to period and to its forward-looking guidance, and to identify operating trends in the company’s principal business.
Non-GAAP financial measures are not meant to be considered in isolation or as a substitute for, or superior to comparable GAAP measures and should be read in conjunction with the consolidated financial information prepared in accordance with GAAP. Investors should note that the Non-GAAP information is not prepared under any comprehensive set of accounting rules or principles and does not reflect all of the amounts associated with the company’s results of operations as determined in accordance with GAAP. Investors should also note that these Non-GAAP financial measures have no standardized meaning prescribed by GAAP and, therefore, have limits in their usefulness to investors. In addition, from time to time in the future there may be other items that the company may exclude for purposes of its Non-GAAP financial measures; likewise, the company may in the future cease to exclude items that it has historically excluded for purposes of its Non-GAAP financial measures. Because of the non-standardized definitions, the Non-GAAP financial measure as used by BioMarin in this press release and the accompanying tables may be calculated differently from, and therefore may not be directly comparable to, similarly titled measures used by other companies.
The following tables present the reconciliation of GAAP reported to Non-GAAP adjusted financial information:
10
Reconciliation of GAAP Reported Information to Non-GAAP Information (1)
(In millions, except percentages and per share data)
(unaudited)
| Three Months Ended September 30, | Nine Months Ended September 30, | ||||||||||||||||||||||
| 2026 | 2025 | 2026 | 2025 | ||||||||||||||||||||
| GAAP Reported Net Income (Loss) | $ | (123) | $ | (31) | $ | 27 | $ | 395 | |||||||||||||||
| Adjustments | |||||||||||||||||||||||
| Stock-based compensation expense - COS | 4 | 4 | 13 | 11 | |||||||||||||||||||
| Stock-based compensation expense - R&D | 14 | 15 | 41 | 41 | |||||||||||||||||||
Stock-based compensation expense - SG&A(2) | 36 | 30 | 119 | 83 | |||||||||||||||||||
| Amortization of intangible assets | 99 | 5 | 177 | 15 | |||||||||||||||||||
Amortization of acquired inventory step-up - COS(3) | 18 | — | 29 | — | |||||||||||||||||||
Acquisition and restructuring-related costs - SG&A(4) | 47 | 15 | 143 | 15 | |||||||||||||||||||
Loss on investments (5) | — | — | — | 3 | |||||||||||||||||||
| Income tax effect of adjustments | (50) | (16) | (120) | (38) | |||||||||||||||||||
| Non-GAAP Income | $ | 45 | $ | 22 | $ | 430 | $ | 525 | |||||||||||||||
| Three Months Ended September 30, | |||||||||||||||||||||||||||||||||||
| 2026 | 2025 | ||||||||||||||||||||||||||||||||||
| COS | R&D | SG&A | COS | R&D | SG&A | ||||||||||||||||||||||||||||||
| GAAP expenses | $ | 205 | $ | 181 | $ | 334 | $ | 140 | $ | 189 | $ | 268 | |||||||||||||||||||||||
| Adjustments | |||||||||||||||||||||||||||||||||||
Stock-based compensation expense(2) | (4) | (14) | (36) | (4) | (15) | (30) | |||||||||||||||||||||||||||||
Amortization of acquired inventory step-up(3) | (18) | — | — | — | — | — | |||||||||||||||||||||||||||||
Acquisition and restructuring-related costs(4) | — | — | (47) | — | — | (15) | |||||||||||||||||||||||||||||
| Non-GAAP expenses | $ | 184 | $ | 167 | $ | 249 | $ | 136 | $ | 174 | $ | 223 | |||||||||||||||||||||||
| Nine Months Ended September 30, | ||||||||||||||||||||||||||||||||||||||
| 2026 | 2025 | |||||||||||||||||||||||||||||||||||||
| COS | R&D | SG&A | COS | R&D | SG&A | |||||||||||||||||||||||||||||||||
| GAAP expenses | $ | 603 | $ | 566 | $ | 987 | $ | 442 | $ | 509 | $ | 707 | ||||||||||||||||||||||||||
| Adjustments | ||||||||||||||||||||||||||||||||||||||
Stock-based compensation expense(2) | (13) | (41) | (119) | (11) | (41) | (83) | ||||||||||||||||||||||||||||||||
Amortization of acquired inventory step-up(3) | (29) | — | — | — | — | — | ||||||||||||||||||||||||||||||||
Acquisition and restructuring-related costs(4) | — | — | (143) | — | — | (15) | ||||||||||||||||||||||||||||||||
| Non-GAAP expenses | $ | 561 | $ | 525 | $ | 724 | $ | 431 | $ | 468 | $ | 609 | ||||||||||||||||||||||||||
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| Three Months Ended September 30, | Nine Months Ended September 30, | |||||||||||||||||||||||||||||||||||||
| 2026 | Percent of GAAP Total Revenue | 2025 | Percent of GAAP Total Revenue | 2026 | Percent of GAAP Total Revenue | 2025 | Percent of GAAP Total Revenue | |||||||||||||||||||||||||||||||
| GAAP Income (Loss) from Operations | $ | (82) | (8.0) | % | $ | (47) | (6.0) | % | $ | 158 | 5.7 | % | $ | 454 | 19.4 | % | ||||||||||||||||||||||
| Adjustments | ||||||||||||||||||||||||||||||||||||||
Stock-based compensation expense(2) | 54 | 5.3 | 49 | 6.3 | 173 | 6.2 | 135 | 5.8 | ||||||||||||||||||||||||||||||
| Amortization of intangible assets | 99 | 9.7 | 5 | 0.6 | 177 | 6.4 | 15 | 0.6 | ||||||||||||||||||||||||||||||
Amortization of acquired inventory step-up(3) | 18 | 1.8 | — | — | 29 | 1.0 | — | — | ||||||||||||||||||||||||||||||
Acquisition and restructuring-related costs(4) | 48 | 4.7 | 15 | 1.9 | 143 | 5.1 | 15 | 0.6 | ||||||||||||||||||||||||||||||
| Non-GAAP Income from Operations | $ | 137 | 13.4 | % | $ | 22 | 2.8 | % | $ | 683 | 24.6 | % | $ | 618 | 26.3 | % | ||||||||||||||||||||||
| Three Months Ended September 30, | Nine Months Ended September 30, | |||||||||||||||||||||||||||||||
| 2026 | 2025 | 2026 | 2025 | |||||||||||||||||||||||||||||
| GAAP Diluted EPS | $ | (0.63) | $ | (0.16) | $ | 0.14 | $ | 2.04 | ||||||||||||||||||||||||
| Adjustments | ||||||||||||||||||||||||||||||||
Stock-based compensation expense(2) | $ | 0.28 | $ | 0.25 | 0.87 | 0.69 | ||||||||||||||||||||||||||
| Amortization of intangible assets | $ | 0.51 | $ | 0.03 | 0.89 | 0.08 | ||||||||||||||||||||||||||
Amortization of acquired inventory step-up(3) | $ | 0.09 | $ | — | 0.15 | — | ||||||||||||||||||||||||||
Acquisition and restructuring-related costs(4) | $ | 0.24 | $ | 0.08 | 0.72 | 0.08 | ||||||||||||||||||||||||||
Loss on investments (5) | $ | — | $ | — | — | 0.02 | ||||||||||||||||||||||||||
| Income tax effect of adjustments | $ | (0.26) | $ | (0.08) | (0.60) | (0.19) | ||||||||||||||||||||||||||
| Non-GAAP Diluted EPS | $ | 0.23 | $ | 0.12 | $ | 2.18 | $ | 2.69 | ||||||||||||||||||||||||
| Three Months Ended September 30, | Nine Months Ended September 30, | ||||||||||||||||||||||
| 2026 | 2025 | 2026 | 2025 | ||||||||||||||||||||
| GAAP Weighted-Average Diluted Shares Outstanding | 193.6 | 192.0 | 195.2 | 196.9 | |||||||||||||||||||
Adjustments(6) | |||||||||||||||||||||||
| Common stock issuable under the company's equity plans | 2.5 | 0.8 | — | — | |||||||||||||||||||
| Common stock issuable under the company’s convertible debt | — | — | 4.4 | — | |||||||||||||||||||
| Non-GAAP Weighted-Average Diluted Shares Outstanding | 196.0 | 192.8 | 199.6 | 196.9 | |||||||||||||||||||
(1) Certain amounts may not sum or recalculate due to rounding.
(2) Stock-based compensation expense recorded in SG&A for the nine months ended September 30, 2026, includes approximately $13 million related to the post-combination service period for unvested Amicus stock options.
(3) Represents amortization of the inventory step-up for the inventory acquired in the Amicus transaction.
(4) The 2026 amounts represent severance, transaction and integration costs resulting from the Amicus acquisition, as well as restructuring costs incurred in connection with corporate initiatives. The 2025 amounts represent severance costs incurred in connection with the acquisition of Inozyme Pharma, Inc.
(5) Represents impairment loss on non-marketable equity securities recorded in Other income, net, in the first quarter of 2025.
(6) Common stock issuable under the company’s equity plans and convertible debt were excluded from the computation of GAAP and Non-GAAP Weighted-Average Diluted Shares Outstanding in the periods they were anti-dilutive.
12