Company Announcements

VOXZOGO is the only approved treatment for children with achondroplasia starting at birth, with over 10 years of clinical research demonstrating the long-term benefit on complications associated with achondroplasia

Researchers will present additional data from studies of VOXZOGO for hypochondroplasia, ahead of pivotal topline Phase 3 data expected in the first half of 2026

SAN RAFAEL, Calif. , May 2, 2026 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced new research from studies of VOXZOGO^® (vosoritide) in children with achondroplasia demonstrating positive impact on important health indicators, including arm span and bone density. The data will be presented, along with new data from studies of VOXZOGO in hypochondroplasia, at the Pediatric Endocrine Society's 2026 Annual Meeting (PES) in San Francisco.

Long-Term Treatment Leads to Meaningful Improvement in Multiple Health Measures

Data from three ongoing long-term extension clinical trials demonstrated the impact of long-term treatment with VOXZOGO on measures beyond height, including arm span and bone health. Researchers showed that arm span Z-scores improved from baseline in all age groups, and the arm span-to-height ratio also remained stable over time, showing treatment resulted in proportional skeletal growth. Children who initiated treatment with VOXZOGO after age 5 also achieved a mean difference in standing height of 10.60 cm after six years of treatment (p<0.0001) and 13.59 cm after eight years of treatment (p<0.0001), compared with untreated natural history cohorts.

"With VOXZOGO, we now have a depth and duration of evidence that is unmatched in the treatment of achondroplasia — providing meaningful insight not only into growth, but into the broader, sustained impact on a child's health," said Bradley Miller, M.D., Ph.D., pediatric endocrinologist at the University of Minnesota Medical School. "When you can see consistent benefits over time, it gives you greater confidence to intervene early and treat with purpose."

Another study with 119 children who received VOXZOGO measured the impact of long-term treatment on bone mineral content (BMC) and bone mineral density (BMD) assessed by dual X-ray absorptiometry (DXA) every year for up to six years. Results showed that BMC increased over time, while BMD Z‑scores remained consistent year over year, demonstrating that bone health was maintained in children who received long-term VOXZOGO treatment.

"We are committed to understanding and sharing the clear impact of long-term treatment with VOXZOGO on health measures beyond height, including arm span and bone density, that are meaningful to the thousands of children around the world receiving this medicine," said Greg Friberg, M.D., Executive Vice President and Chief Research & Development Officer at BioMarin. "With more than ten years of clinical research now, we have seen again and again a breadth of data that continue to demonstrate that long-term and early treatment are critical to ensure the greatest benefit in children with achondroplasia."

New Research in Hypochondroplasia; Topline Phase 3 Results Expected in 1H 2026

Two studies focused on VOXZOGO in hypochondroplasia will also be presented, including one from a single-arm Phase 2 study conducted by Children's National Hospital that showed a statistically significant improvement in total body minus head BMD of 0.03 g/cm² (p<0.0001) and BMC of 54.84 g (p<0.0001) after 12 months in children who received the medicine.

The company plans to share topline results from its registration-enabling Phase 3 pivotal clinical trial of VOXZOGO in children with hypochondroplasia (CANOPY-HCH-3) in the first half of 2026. If the clinical results are positive, the company plans to submit data to health authorities in the second half of 2026 to seek approval for this new indication.

Below are key poster presentations for achondroplasia and hypochondroplasia at PES, with all times listed in Pacific Daylight Time:

Design of a Randomized, Double-Blind, Placebo-Controlled Phase 2 Study to Evaluate the Safety and Efficacy of Vosoritide in Infants and Children With Hypochondroplasia Aged <3 Years
Poster #12, Poster Session 2
Friday, May 1, 7:15 – 8:15 a.m.

Effect of Long-Term Vosoritide Treatment in Pediatric Participants With Achondroplasia on Bone Mineral Density and Bone Mineral Content Measured with Dual X-Ray Absorptiometry
Poster #16, Poster Session 3
Friday, May 1, 12:30 – 2 p.m.

Real-World Long-Term Effectiveness and Safety Outcomes of Vosoritide in Adolescents With Achondroplasia in Japan
Poster #58, Poster Session 3
Friday, May 1, 12:30 – 2 p.m.

Vosoritide Treatment Improves Linear Growth and Absolute Bone Content and Density in Children With Hypochondroplasia: A 12-Month Prospective Study
Poster #40, Poster Session 3
Friday, May 1, 12:30 – 2 p.m.

Effect of Long-Term Vosoritide Treatment on Growth in Children With Achondroplasia in Open-Label, Multicenter Clinical Trials
Poster #32, Poster Session 5
Saturday, May 2, 12:30 – 2 p.m.

Improving Guideline-Directed Management of Achondroplasia: Results of a Pre-Implementation Study
Poster #12, Poster Session 5
Saturday, May 2, 12:30 – 2 p.m.

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

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 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 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 the Pediatric Endocrine Society's 2026 Annual Meeting, including the key poster presentations; the development of BioMarin's VOXZOGO program, including plans to share topline results from the Phase 3 pivotal clinical trial of VOXZOGO in children with hypochondroplasia (CANOPY-HCH-3) in the first half of 2026 and to submit data to health authorities in the second half of 2026 if such results are positive; the safety profile and potential benefits of VOXZOGO for children with achondroplasia and hypochondroplasia; and the continued clinical development of VOXZOGO in multiple indications. 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, 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 Annual Report on Form 10-K for the year ended December 31, 2025, 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.

 

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