BioMarin Announces Positive Results of Phase 3 VOXZOGO® (vosoritide) Study in Hypochondroplasia Published in NEJM Evidence

Phase 3 VOXZOGO data in hypochondroplasia met the primary growth endpoint with a safety profile consistent with prior achondroplasia studies.

Rhea-AI Summary

BioMarin (BMRN) reported that Phase 3 CANOPY‑HCH‑3 data for VOXZOGO (vosoritide) in children with hypochondroplasia were published in NEJM Evidence and presented at the 2026 ESPE meeting.

Treatment with VOXZOGO met the study’s primary endpoint, showing a statistically significant improvement in annualized growth velocity versus placebo after 52 weeks, with an LS mean difference of 2.33 cm/year (p<0.0001). VOXZOGO also improved standing height (LS mean difference 2.35 cm; p<0.0001), height Z‑score (LS mean difference 0.39 standard deviation score; p<0.0001) and arm span (LS mean difference 1.03 cm; p=0.0082). Quality‑of‑life measures showed numerical improvements and longer‑term follow‑up is ongoing.

The safety profile was consistent with prior VOXZOGO studies in achondroplasia, with most adverse events mild and no treatment‑related serious events identified. BioMarin has submitted an sNDA to the FDA, is preparing submissions to EMA and other regulators, and targets a potential 2027 launch if approved.

Positive

• Annualized growth velocity LS mean difference +2.33 cm/year vs placebo at 52 weeks (p<0.0001)
• Standing height LS mean difference +2.35 cm vs placebo at 52 weeks (p<0.0001)
• Height Z‑score LS mean difference +0.39 vs placebo at 52 weeks (p<0.0001)
• Arm span LS mean difference +1.03 cm vs placebo at 52 weeks (p=0.0082)
• No treatment‑related serious adverse events observed; most adverse events were mild
• sNDA submitted to FDA for VOXZOGO in hypochondroplasia; potential 2027 launch if approved

Negative

• None.

Market Context

Before publication, BMRN was down 2.86%; the release detailed the same CANOPY-HCH-3 program previous...

Analysis

Before publication, BMRN was down 2.86%; the release detailed the same CANOPY-HCH-3 program previously reported on May 20, linking the NEJM Evidence publication and sNDA submission to an established Phase 3 record.

Key Figures

Annualized growth velocity: 2.33 cm/year; p<0.0001 Standing height: 2.35 cm; p<0.0001 Height Z-score: 0.39 standard deviation score; p<0.0001 +2 more

Annualized growth velocity

2.33 cm/year; p<0.0001

52-week Phase 3 CANOPY-HCH-3 study versus placebo

Standing height

2.35 cm; p<0.0001

52-week Phase 3 CANOPY-HCH-3 study versus placebo

Height Z-score

0.39 standard deviation score; p<0.0001

52-week Phase 3 CANOPY-HCH-3 study versus placebo

Arm span

1.03 cm; p=0.0082

52-week Phase 3 CANOPY-HCH-3 study versus placebo

Potential launch

2027

If VOXZOGO is approved for hypochondroplasia

Previous Clinical trial Reports

1 past event · Latest: May 20

Same Type 1 event

1. May 20

   Phase 3 clinical data

   24h Move

   +7.8%

   Reported the same CANOPY-HCH-3 endpoint and planned FDA submission for hypochondroplasia

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

Key Terms

snda, annualized growth velocity, height z-score, pdufa

4 terms

snda regulatory

"submitted a supplemental New Drug Application (sNDA) to the FDA"

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.

annualized growth velocity medical

"statistically significant improvements in annualized growth velocity"

Annualized growth velocity is a way of expressing how fast a financial metric (such as revenue, earnings, or user base) would grow over a full year based on growth observed during a shorter period. Investors use it to compare performance across companies or timeframes and to project future size; think of timing how far a car travels in one minute and converting that to a miles‑per‑hour estimate to understand overall speed.

height z-score medical

"standing height, height Z-score and arm span after 52 weeks"

A height z-score is a standardized number that shows how an individual’s height compares to a reference population, measured in units of standard deviation. Think of it like saying someone is two “steps” above or below the average height for their age and sex. Investors care because it’s a common, objective measure used in clinical trials, product labeling and patient eligibility for growth-related therapies, so changes in z-scores can affect regulatory decisions, market size estimates and treatment value.

pdufa regulatory

"FDA Prescription Drug User Fee Act (PDUFA) target action date"

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.

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

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Detailed Phase 3 CANOPY-HCH-3 data in children living with hypochondroplasia also featured in a late-breaking oral presentation at the European Society for Paediatric Endocrinology (ESPE) 2026 Annual Meeting

VOXZOGO demonstrated statistically significant improvements across multiple measures of growth, including annualized growth velocity, standing height, height Z-score and arm span

BioMarin recently submitted a supplemental New Drug Application (sNDA) to the FDA to support expanding treatment with VOXZOGO to include children with hypochondroplasia

SAN RAFAEL, Calif., Sept. 9, 2026 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced new data from the Phase 3 CANOPY-HCH-3 study evaluating VOXZOGO^® (vosoritide) in children with hypochondroplasia were published in New England Journal of Medicine (NEJM) Evidence and presented at the European Society for Paediatric Endocrinology (ESPE) 2026 Annual Meeting. The data included new results on the magnitude of benefit seen in children receiving VOXZOGO, including statistically significant improvements in annualized growth velocity (AGV), standing height, height Z-score and arm span after 52 weeks, with safety findings consistent with the established profile of VOXZOGO in achondroplasia.

The CANOPY-HCH-3 study showed that treatment with VOXZOGO led to a statistically significant improvement in AGV compared with placebo after 52 weeks, meeting the study's primary endpoint (least squares [LS] mean difference of 2.33 cm/year; p<0.0001). Children treated with VOXZOGO also showed significant improvements in standing height (LS mean difference of 2.35 cm; p<0.0001), height Z-score (LS mean difference of 0.39 standard deviation score; p<0.0001), and arm span (LS mean difference of 1.03 cm; p=0.0082) compared with placebo. Children who received VOXZOGO also demonstrated numerical improvements in quality of life, and follow-up will continue to assess the impact of treatment over a longer term. The overall safety profile was consistent with previous studies of VOXZOGO, with most adverse events reported as mild and no treatment-related serious adverse events identified.

"These results presented in detail for the first time provide a comprehensive picture of the impact of VOXZOGO across multiple measures of growth in children with hypochondroplasia," said Greg Friberg, M.D., Executive Vice President and Chief Research & Development Officer at BioMarin. "Based on this compelling body of evidence, we have submitted these data to the FDA with the goal of securing approval for the first medicine for children with hypochondroplasia."

"Hypochondroplasia can affect a child's growth, physical function and everyday life, with families often navigating unique challenges as they support their children's development," said Andrew Dauber, M.D., lead study investigator and Chief of Endocrinology at Children's National in Washington, D.C. "The changes we observed in annualized growth velocity and arm span provide encouraging evidence of how children with hypochondroplasia responded to treatment throughout the study. These findings deepen our understanding of the condition while reinforcing VOXZOGO's potential as the first targeted medicine developed specifically for children with hypochondroplasia."

BioMarin recently submitted its supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for the approval of VOXZOGO for the treatment of hypochondroplasia and are on track with the submissions to the European Medicines Agency (EMA) and other regional health authorities. If approved, VOXZOGO would be the first targeted therapy for the treatment of hypochondroplasia, with a potential 2027 launch.

Below are key BioMarin presentations across both achondroplasia and hypochondroplasia at ESPE, with all times listed in Central European Summer Time:

Vosoritide Increases Growth Velocity in Children With Hypochondroplasia: Phase 3 Trial Results
Oral Presentation #LBA 1067
Wednesday, Sept. 9, 10:48 – 10:56 a.m.

Vosoritide Safety and Effectiveness in Young Children With Achondroplasia Aged ≤3 Years and With up to 36 Months of Follow-Up from the Japanese Post-Marketing Safety Surveillance Study (111-604)
Oral Presentation #FC3.4
Tuesday, Sept. 8, 3:30 – 3:40 p.m.

About Hypochondroplasia 

Hypochondroplasia is a rare, genetic skeletal dysplasia characterized by impaired bone growth, leading to disproportionate short stature and skeletal differences that can affect the long bones, spine and other parts of the skeleton and may impact physical functioning and overall quality of life. The condition presents with a broad and variable clinical spectrum and may include otolaryngologic (related to the ears, nose and throat) and neurological complications and is often diagnosed in toddlerhood or early school age based on clinical and radiological findings. BioMarin estimates that roughly 14,000 children with hypochondroplasia within the company's global footprint may be eligible for treatment with VOXZOGO. 

There are currently no medicines approved by the U.S. Food and Drug Administration or the European Medicines Agency for the treatment of hypochondroplasia. 

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

About VOXZOGO 

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). An sNDA with long-term safety and efficacy data from three ongoing studies, including adult height and additional clinical outcomes beyond linear growth such as body proportionality and arm span is under review with an FDA Prescription Drug User Fee Act (PDUFA) target action date of Feb. 28, 2027.

The use of VOXZOGO to treat hypochondroplasia has not yet been approved by any regulatory agency. 

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: the data to be presented at European Society for Paediatric Endocrinology (ESPE) 2026 Annual Meeting, including the safety profile and potential benefits of VOXZOGO for children with hypochondroplasia and achondroplasia; BioMarin's plans and expectations for the development of VOXZOGO for children with hypochondroplasia, including the expectation that, if approved by the U.S. Food and Drug Administration (FDA), VOXZOGO would be the first targeted therapy for the treatment of hypochondroplasia with a potential 2027 launch; BioMarin's expectations regarding its supplemental New Drug Application (sNDA) for VOXZOGO for full approval in children with achondroplasia, including expectations regarding the Prescription Drug User Fee Act (PDUFA) target action date; and BioMarin's estimate regarding total addressable patient population (TAPP) with respect to the conditions targeted by BioMarin's product candidates and commercial products, including hypochondroplasia. 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 and the release of data from those trials; any potential adverse events observed in the continuing monitoring of the patients in the clinical trials; the content and timing of decisions by the FDA, 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 June 30, 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^® 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 was the primary endpoint in the CANOPY‑HCH‑3 Phase 3 study and how did VOXZOGO perform?

The primary endpoint was the difference in annualized growth velocity after 52 weeks between VOXZOGO and placebo. VOXZOGO achieved a statistically significant improvement, with a least squares mean difference of 2.33 cm/year compared with placebo (p<0.0001), thereby meeting the primary endpoint.

What additional growth and body proportion measures improved with VOXZOGO treatment?

Children treated with VOXZOGO showed statistically significant improvements in standing height (LS mean difference 2.35 cm; p<0.0001), height Z‑score (LS mean difference 0.39 standard deviation score; p<0.0001), and arm span (LS mean difference 1.03 cm; p=0.0082) versus placebo after 52 weeks.

What safety results were observed for VOXZOGO in children with hypochondroplasia?

The overall safety profile of VOXZOGO in the CANOPY‑HCH‑3 study was consistent with its established profile in achondroplasia. Most adverse events were reported as mild, and no treatment‑related serious adverse events were identified.

What is BioMarin’s regulatory and launch timeline for VOXZOGO in hypochondroplasia?

BioMarin has submitted a supplemental New Drug Application (sNDA) to the U.S. FDA to seek approval of VOXZOGO for hypochondroplasia and is on track with submissions to the EMA and other regional authorities. The company indicates that, if approved, VOXZOGO could launch for hypochondroplasia in 2027.

How many children with hypochondroplasia might be eligible for VOXZOGO treatment within BioMarin’s footprint?

BioMarin estimates that roughly 14,000 children with hypochondroplasia within its global footprint may be eligible for treatment with VOXZOGO, if approved for this indication.

Are there currently any approved medicines for hypochondroplasia?

There are no medicines currently approved by the U.S. Food and Drug Administration or the European Medicines Agency for the treatment of hypochondroplasia. VOXZOGO for hypochondroplasia remains investigational and has not been approved by any regulatory agency.

What ongoing regulatory review is mentioned for VOXZOGO in achondroplasia?

An sNDA containing long‑term safety and efficacy data for VOXZOGO in achondroplasia, including adult height and additional outcomes such as body proportionality and arm span, is under review by the FDA with a Prescription Drug User Fee Act (PDUFA) target action date of February 28, 2027.