U.S. Food and Drug Administration Accepts BioMarin's Supplemental New Drug Application for Full Approval of VOXZOGO® (vosoritide) for Children With Achondroplasia

Rhea-AI Summary

BioMarin (Nasdaq:BMRN) announced that the U.S. FDA has accepted its supplemental New Drug Application (sNDA) seeking full approval of VOXZOGO (vosoritide) for children with achondroplasia, and set a Prescription Drug User Fee Act (PDUFA) target action date of February 28, 2027.

According to BioMarin, the sNDA is supported by long-term safety and efficacy data from three ongoing studies (111-205, 111-208, 111-302), including adult height and additional clinical outcomes such as body proportionality and arm span. The submission is intended to fulfill the postmarketing requirement for converting VOXZOGO’s 2021 accelerated approval to traditional approval, backed by what the company describes as the longest efficacy and safety data package for a medicine studied in achondroplasia.

Positive

• FDA acceptance of VOXZOGO sNDA with PDUFA date February 28, 2027
• sNDA supported by long-term data from three ongoing studies (111-205, 111-208, 111-302)
• Data include adult height and multiple skeletal growth measures beyond linear growth
• Submission aims to convert 2021 accelerated approval to traditional approval
• Company reports longest efficacy and safety dataset for any medicine in achondroplasia

Negative

• Regulatory decision timeline extends to at least February 28, 2027
• Full approval still contingent on FDA review of long-term safety and efficacy data

News Explained

FDA acceptance advances VOXZOGO’s full-approval application, but the Feb. 28, 2027 target date is not completed approval.

The July 13, 2026 release reports that the FDA accepted BioMarin’s supplemental application for full approval of VOXZOGO, while describing the conversion as conditional on a later approval decision.

For the company, the disclosed change is a regulatory-lifecycle milestone: the full-approval application has been accepted, but the release does not report completion of the conversion from accelerated approval to traditional approval.

The release defines accelerated approval as a pathway enabling faster patient access based on measures reasonably likely to predict clinical benefit, and says the supplemental application is intended to fulfill the postmarketing requirement confirming that benefit.

The submission is supported by long-term safety and efficacy data from three ongoing studies, including adult-height, body-proportionality, and other clinical outcomes.

The next named milestone is the FDA’s PDUFA target action date of Feb. 28, 2027; that is a target action date rather than a reported approval date.

News Market Reaction – BMRN

+0.14%

+0.14% Session close to close

In the Jul 13 session, BMRN gained 0.14%, reflecting a mild positive market reaction.

Data tracked by StockTitan Argus on the day of publication.

Market Context

FDA acceptance of VOXZOGO’s supplemental application with a Feb 28, 2027 PDUFA date strengthens the ...

Analysis

FDA acceptance of VOXZOGO’s supplemental application with a Feb 28, 2027 PDUFA date strengthens the achondroplasia franchise, supported by three long-term studies. Investors may weigh this against mixed past news reactions and recent insider net selling when assessing durability of the opportunity.

Key Figures

PDUFA target date: Feb 28, 2027 Supporting studies: 3 ongoing studies Study identifiers: 111-205, 111-208, 111-302 +1 more

4 metrics

PDUFA target date Feb 28, 2027 FDA action date for VOXZOGO full-approval sNDA

Supporting studies 3 ongoing studies Long-term safety and efficacy data for VOXZOGO sNDA

Study identifiers 111-205, 111-208, 111-302 Trials providing long-term VOXZOGO data in achondroplasia

Initial FDA approval year 2021 Year VOXZOGO received FDA accelerated approval

Historical Context

5 past events · Latest: Jun 16 (Positive)

Pattern 5 events

Date	Event	Sentiment	24h Move	Catalyst
Jun 16	VOXZOGO data update	Positive	-0.9%	New three-year VOXZOGO and BMN 333 data presented at ENDO 2026.
May 20	VOXZOGO Phase 3 data	Positive	+7.8%	Positive Phase 3 CANOPY-HCH-3 results in hypochondroplasia, primary endpoint met.
May 18	BMN 401 Phase 3 update	Negative	-4.1%	Phase 3 trial met biochemical endpoint but missed key clinical efficacy measures.
May 05	Financing and board changes	Neutral	-4.0%	4D Path announced strategic financing and added two directors to its board.
May 04	Q1 2026 earnings	Neutral	-4.0%	Q1 revenues reported with higher 2026 guidance but lower GAAP income and EPS.

24h Move is the share-price change in the day after each event; other market factors may also have contributed.

Pattern Detected

Recent news has produced mixed reactions, with only one VOXZOGO Phase 3 update driving a strong positive move.

Key Terms

supplemental new drug application, snda, prescription drug user fee act, pdufa, +2 more

6 terms

supplemental new drug application regulatory

"the company's supplemental New Drug Application (sNDA) for VOXZOGO"

A supplemental new drug application is a request submitted to regulatory authorities to make changes to an existing approved medication, such as adding new uses, strengths, or formulations. For investors, it signals that a pharmaceutical company is seeking approval for new product developments or expanded applications, which can impact the company's future sales, market potential, and stock value.

snda regulatory

"The sNDA submission was supported by substantial long-term safety and efficacy data"

A SNDA (Subordination, Non‑Disturbance and Attornment Agreement) is a legal pact among a property owner’s lender, the owner’s tenants, and sometimes the landlord that sets who keeps lease rights if the property is sold or a mortgage is enforced. Think of it as a rulebook that decides whether a tenant can stay and keep paying rent or must answer to a new owner after a foreclosure. For investors, an SNDA matters because it protects predictable rental income, clarifies who has priority on claims against a property, and therefore affects a property’s value and the security of related loans.

prescription drug user fee act regulatory

"The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date"

A federal program that lets drug makers pay fees to the U.S. regulator to fund and speed up the review of new medicines and label changes. Investors care because it affects how quickly a drug can move from testing to market and how predictable approval timelines and regulatory interactions are — like buying a faster lane at a busy checkpoint that can reduce uncertainty about a product’s commercial timing.

pdufa regulatory

"FDA PDUFA target action date of Feb."

PDUFA is the Prescription Drug User Fee Act, the U.S. law under which drug companies pay fees that fund the FDA's review of new medicines. In company news the term usually appears as the PDUFA date, the target deadline by which the FDA aims to decide on a drug application; that date tells investors when to expect the approval or rejection decision for the product.

accelerated approval regulatory

"VOXZOGO received FDA accelerated approval in 2021"

Accelerated approval is a process that allows new medical treatments to be approved more quickly than usual if they address serious or life-threatening conditions and show promising early results. For investors, it signals that a treatment may reach the market sooner, potentially boosting a company's prospects, but it also involves some uncertainty since full evidence of effectiveness is still being gathered.

postmarketing requirement regulatory

"This sNDA is intended to fulfill the postmarketing requirement to confirm that benefit"

A postmarketing requirement is a study or set of studies that a regulatory agency orders after a drug or medical device is approved to collect more data on safety, effectiveness, dosing, or long-term risks. Think of it like an official follow-up test required after a product launch to answer questions that initial trials didn’t fully resolve. Results can affect labeling, market access, and public confidence, so they matter to investors monitoring regulatory and commercial risk.

AI-generated analysis. How Rhea-AI works. Not financial advice.

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Application based on long-term safety and efficacy data from three ongoing studies, including adult height and additional clinical outcomes beyond linear growth, including body proportionality and arm span evaluated over long-term follow-up

FDA PDUFA target action date of Feb. 28, 2027

SAN RAFAEL, Calif., July 13, 2026 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the U.S. Food and Drug Administration (FDA) has accepted the company's supplemental New Drug Application (sNDA) for VOXZOGO^® (vosoritide) for full approval in children with achondroplasia. The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of Feb. 28, 2027.

"This submission for VOXZOGO is supported by the largest body of evidence for any medicine in achondroplasia, reflecting BioMarin's long-standing commitment to advancing the science of skeletal growth. The clinical data demonstrate meaningful improvements across multiple skeletal growth-related measures beyond annualized growth velocity in children with this condition," said Greg Friberg, M.D., Executive Vice President and Chief Research & Development Officer at BioMarin. "If approved, VOXZOGO would be the first therapy for achondroplasia to convert from accelerated approval to traditional approval based on a comprehensive clinical data package, including adult height outcomes and other clinical measures evaluated over extended follow-up."

The sNDA submission was supported by substantial long-term safety and efficacy data from three ongoing studies (111-205, 111-208 and 111-302), including clinically meaningful results in growth and improvements across key skeletal growth-related measures, including proportionality and arm span. The full package submitted to the FDA included the longest efficacy and safety data of any medicine studied in achondroplasia.

VOXZOGO received FDA accelerated approval in 2021, a pathway enabling faster patient access based on measures reasonably likely to predict clinical benefit. This sNDA is intended to fulfill the postmarketing requirement to confirm that benefit and convert to full approval, supported by long-term data from three ongoing studies demonstrating clinically meaningful improvements in growth and skeletal health outcomes in children with achondroplasia.

About Achondroplasia

Achondroplasia, the most common form of skeletal dysplasia leading to disproportionate short stature in humans, is characterized by slowing of endochondral ossification, which results in disproportionate short stature and disordered architecture in the long bones, spine, face and base of the skull. This condition is caused by a change in the FGFR3 gene, a negative regulator of bone growth.

More than 80% of children with achondroplasia have parents of average stature and have the condition as the result of a spontaneous gene mutation. The worldwide incidence rate of achondroplasia is about one in 25,000 live births. VOXZOGO is being tested in children whose growth plates are still "open," typically those under 18 years of age. Approximately 25% of people with achondroplasia fall into this category.

For more information about our clinical trials in achondroplasia, hypochondroplasia and other skeletal conditions, please visit clinicaltrials.biomarin.com.

About VOXZOGO (vosoritide)

In children with achondroplasia, endochondral bone growth, an essential process by which bone tissue is created, is negatively regulated due to a gain of function mutation in FGFR3. VOXZOGO, a C-type natriuretic peptide (CNP) analog, acts as a positive regulator of the signaling pathway downstream of FGFR3 to promote endochondral bone growth.

VOXZOGO is the only approved medicine to support the growth of children with achondroplasia starting from birth, with international consensus guidelines recommending initiation of VOXZOGO as early as possible. First approved in 2021, VOXZOGO has helped more than 5,000 infants and children in more than 50 countries. Through our ongoing studies, BioMarin continues to evaluate VOXZOGO on key clinical endpoints relevant for achondroplasia patients, such as arm span, tibial bowing (leg bowing), body proportionality, spinal morphology (including spinal stenosis) and quality of life measures.

VOXZOGO is approved in the U.S., Japan and Australia to increase linear growth in children of all ages with achondroplasia with open epiphyses, and VOXZOGO is indicated in the EU for the treatment of achondroplasia in children 4 months of age and older whose epiphyses are not closed, as confirmed by appropriate genetic testing. In the U.S., this indication is approved under accelerated approval based on an improvement in annualized growth velocity. Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trial(s). To fulfill this post-marketing requirement, BioMarin intends to use the ongoing open-label extension studies compared to available natural history.

Patient Support Accessing VOXZOGO

BioMarin's robust support services have ensured a seamless treatment experience, spearheaded by Clinical Coordinators, who have conducted hundreds of trainings for families with achondroplasia since approval. BioMarin provides resources to support families navigating achondroplasia, including a caregiver mentorship program that connects parents with other caregivers, and a U.S. doctor directory that helps families and healthcare professionals identify clinicians experienced in achondroplasia care.

To reach a BioMarin RareConnections^® Case Manager, please call, toll-free, 1-833-VOXZOGO (1-833-869-9646) or e-mail VOXZOGOSupport@biomarin-rareconnections.com. For more information about VOXZOGO, please visit www.voxzogo.com. For additional information regarding this product, please contact BioMarin Medical Information at medinfo@bmrn.com.

VOXZOGO U.S. Important Safety Information

What is VOXZOGO used for? 

• VOXZOGO is a prescription medicine used to increase linear growth in children with achondroplasia and open growth plates (epiphyses). 
• VOXZOGO is approved under accelerated approval based on an improvement in annualized growth velocity. Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trials. 

What is the most important safety information about VOXZOGO? 

• VOXZOGO may cause serious side effects including a temporary decrease in blood pressure in some patients. To reduce the risk of a decrease in blood pressure and associated symptoms (dizziness, feeling tired, or nausea), patients should eat a meal and drink 8 to 10 ounces of fluid within 1 hour before receiving VOXZOGO. 

What are the most common side effects of VOXZOGO? 

• The most common side effects of VOXZOGO include injection site reactions (including redness, itching, swelling, bruising, rash, hives, and injection site pain), high levels of blood alkaline phosphatase shown in blood tests, vomiting, joint pain, decreased blood pressure, and stomachache. These are not all the possible side effects of VOXZOGO. Ask your healthcare provider for medical advice about side effects, and about any side effects that bother the patient or that do not go away. 

How is VOXZOGO taken? 

• VOXZOGO is taken daily as an injection given under the skin, administered by a caregiver after a healthcare provider determines the caregiver is able to administer VOXZOGO. Do not try to inject VOXZOGO until you have been shown the right way by your healthcare provider. VOXZOGO is supplied with Instructions for Use that describe the steps for preparing, injecting, and disposing VOXZOGO. Caregivers should review the Instructions for Use for guidance and any time they receive a refill of VOXZOGO in case any changes have been made. 
• Inject VOXZOGO 1 time every day, at about the same time each day. If a dose of VOXZOGO is missed, it can be given within 12 hours from the missed dose. After 12 hours, skip the missed dose and administer the next daily dose as usual. 
• The dose of VOXZOGO is based on body weight. Your healthcare provider will adjust the dose based on changes in weight following regular check-ups. 
• Your healthcare provider will monitor the patient's growth and tell you when to stop taking VOXZOGO if they determine the patient is no longer able to grow. Stop administering VOXZOGO if instructed by your healthcare provider. 

What should you tell the doctor before or during taking VOXZOGO? 

• Tell your doctor about all of the patient's medical conditions including 
  • If the patient has heart disease (cardiac or vascular disease), or if the patient is on blood pressure medicine (anti-hypertensive medicine). 
  • If the patient has kidney problems or renal impairment. 
  • If the patient is pregnant or plans to become pregnant. It is not known if VOXZOGO will harm the unborn baby. 
  • If the patient is breastfeeding or plans to breastfeed. It is not known if VOXZOGO passes into breast milk. 

• Tell your doctor about all of the medicines the patient takes, including prescription and over-the-counter medicines, vitamins, and herbal supplements. 

You may report side effects to BioMarin at 1-866-906-6100. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088. 

Please see additional safety information in the full Prescribing Information and Patient Information. 

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.

Forward-Looking Statements

This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including without limitation, statements about: BioMarin's expectations regarding the submission of its supplemental New Drug Application (sNDA) for VOXZOGO (vosoritide) for full approval in children with achondroplasia, including expectations regarding the Prescription Drug User Fee Act (PDUFA) target action date; the safety profile and potential benefits of VOXZOGO for children with achondroplasia, including benefits beyond height; and the development of BioMarin's VOXZOGO program generally and the continued clinical development of VOXZOGO, including in achondroplasia, hypochondroplasia and other skeletal conditions. 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: results and timing of current and planned preclinical studies and clinical trials of VOXZOGO; any potential adverse events observed in the continuing monitoring of the patients in the clinical trials; 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; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission (SEC), including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended March 31, 2026, as such factors may be updated by any subsequent filings with the SEC. 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^®, BioMarin RareConnections^® and VOXZOGO^® are registered trademarks of BioMarin Pharmaceutical Inc.

Contacts:
Investors	Media
Traci McCarty	Andrew Villani
BioMarin Pharmaceutical Inc.	BioMarin Pharmaceutical Inc.
(415) 455-7558	(628) 269-7393

 

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SOURCE BioMarin Pharmaceutical Inc.

FAQ

What did the FDA accept for BioMarin’s VOXZOGO (BMRN) in July 2026?

The FDA accepted BioMarin’s supplemental New Drug Application for full approval of VOXZOGO for children with achondroplasia. According to BioMarin, this filing is based on extensive long-term safety and efficacy data from three ongoing clinical studies in this patient population.

What is the FDA PDUFA action date for BioMarin’s VOXZOGO sNDA (BMRN)?

The FDA set a Prescription Drug User Fee Act (PDUFA) target action date of February 28, 2027 for VOXZOGO’s sNDA. According to BioMarin, this date marks when the agency is expected to complete its review for potential full approval.

What clinical data support BioMarin’s VOXZOGO full approval request for achondroplasia (BMRN)?

The VOXZOGO sNDA is supported by long-term safety and efficacy data from three ongoing studies: 111-205, 111-208 and 111-302. According to BioMarin, these data include adult height, body proportionality, arm span, and other skeletal growth-related outcomes over extended follow-up.

How would full FDA approval of VOXZOGO (BMRN) differ from its current accelerated approval?

VOXZOGO currently has FDA accelerated approval granted in 2021, based on measures reasonably likely to predict benefit. According to BioMarin, the sNDA aims to convert this to traditional approval by fulfilling postmarketing requirements with confirmed long-term growth and skeletal health outcomes.

What patient outcomes were evaluated in VOXZOGO’s long-term achondroplasia studies for BioMarin (BMRN)?

The long-term studies evaluated annualized growth, adult height, and skeletal growth-related measures such as body proportionality and arm span. According to BioMarin, these outcomes showed clinically meaningful improvements in children with achondroplasia over extended follow-up periods.

Why is BioMarin’s VOXZOGO data package for achondroplasia considered significant for BMRN investors?

BioMarin describes the VOXZOGO submission as the largest body of evidence for any medicine in achondroplasia. According to BioMarin, it includes the longest efficacy and safety dataset, which underpins the company’s request to convert to full FDA approval.