BioMarin Announces Positive Phase 3 Pivotal Study Results for VOXZOGO® (vosoritide) in Children with Hypochondroplasia

Study met primary endpoint, exceeding expectations with a highly statistically significant improvement in annualized growth velocity (AGV, change from baseline) of 2.33 cm/yr compared to placebo at week 52, as well as statistically significant increases in standing height and height Z-score

Study demonstrated statistically significant improvement in arm span at week 52 compared to placebo, a key measure linked to functional independence

Safety findings consistent with the established profile in achondroplasia with no new safety signals identified

Supplemental New Drug Application (sNDA) submission to U.S. Food and Drug Administration (FDA) planned for third quarter, followed by submissions to the European Medicines Agency (EMA) and other regional health authorities

Full data to be presented at an upcoming medical meeting

SAN RAFAEL, Calif., May 20, 2026 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the Phase 3 CANOPY-HCH-3 study evaluating treatment with VOXZOGO^® (vosoritide) in children with hypochondroplasia met its primary endpoint, demonstrating a statistically significant increase in the change from baseline at week 52 in annualized growth velocity (AGV) compared to placebo (LS mean difference +2.33 cm/yr, p<0.0001). Children who received VOXZOGO also showed a statistically significant increase in standing height (p<0.0001) and height Z-score (p<0.0001) versus placebo after one year of treatment.

Additionally, significant improvements in arm span were demonstrated (p=0.004), a key prespecified secondary endpoint in the study. These positive findings could have important implications for children with hypochondroplasia, offering improvements in reach, daily activities and independence, which have been highlighted as meaningful outcomes by the community.

"As someone who treats children with hypochondroplasia, I find these positive results tremendously encouraging. Seeing these improvements in growth is a milestone we have hoped for after so many years without treatment options," said Dr. Andrew Dauber, who is the lead study investigator and Chief of Endocrinology at Children's National Hospital in Washington, D.C. "These data suggest we may be approaching a new era in how we care for children with hypochondroplasia."

"For the first time, a pivotal Phase 3 study in hypochondroplasia has demonstrated impressive gains in growth, including improvements in arm span, highlighting VOXZOGO's potential to fundamentally change the treatment landscape for this condition," said Greg Friberg, M.D., Executive Vice President and Chief Research & Development Officer at BioMarin. "We are incredibly pleased with these results, which exceeded our expectations for this study, and we are deeply grateful to the families and investigators who made this outcome possible. We look forward to sharing these data with regulatory authorities and the broader community as we work to bring this medicine to children living with hypochondroplasia around the world."

The safety findings in the study for VOXZOGO were consistent with the established profile in achondroplasia and no new safety signals were observed. Full results from the CANOPY-HCH-3 study will be presented at an upcoming medical meeting.

The supplemental New Drug Application (sNDA) submission to the U.S. Food and Drug Administration (FDA) is planned for the third quarter of 2026, followed by submissions to the European Medicines Agency (EMA) and other regional health authorities.

About CANOPY-HCH-3

CANOPY-HCH-3 (Study 111-303) is a global Phase 3, 1:1 randomized, double-blind, placebo-controlled, multicenter study designed to evaluate the efficacy and safety of VOXZOGO in 80 children ages 3 to 17 with hypochondroplasia. The primary endpoint of the study was change from baseline in annualized growth velocity (AGV) at 52 weeks compared to placebo. Key secondary pre-specified endpoints included changes from baseline in standing height, height Z-score, arm span, upper to lower body segment ratio, and health-related quality of life, along with assessments of safety and tolerability. Study participants will continue to be followed in a long-term extension study.

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.

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 (vosoritide)

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 submitted its U.S. supplemental New Drug Application (sNDA) for full approval of VOXZOGO for achondroplasia in April 2026, and the company expects to be notified of sNDA acceptance by the third quarter of 2026.

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 https://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 a portfolio of 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 https://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 development of BioMarin's VOXZOGO program generally, and the results of the pivotal Phase 3 CANOPY-HCH-3 study evaluating treatment with VOXZOGO in children with hypochondroplasia particularly; the safety profile and potential benefits of VOXZOGO for children with hypochondroplasia, including VOXZOGO's potential to fundamentally change the treatment landscape for hypochondroplasia; BioMarin's plans to submit for regulatory approval to global health authorities in the second half of 2026; and BioMarin's expectations regarding the demand for VOXZOGO. 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, the final analysis of the results from CANOPY-HCH-3, 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^® and VOXZOGO^® are registered trademarks of BioMarin Pharmaceutical Inc.

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SOURCE BioMarin Pharmaceutical Inc.