BioMarin Presents New Data Advancing Understanding of Achondroplasia and Hypochondroplasia Treatment at the American Society for Bone and Mineral Research Annual Meeting

New analysis showcases impact of VOXZOGO^® (vosoritide) in foramen magnum and craniocervical junction growth in children with achondroplasia, including infants

SAN RAFAEL, Calif., Oct. 9, 2026 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced it will present new data from 13 studies at the American Society for Bone and Mineral Research 2026 Annual Meeting (ASBMR) in Boston, including results from studies of VOXZOGO^® (vosoritide) in children with achondroplasia and hypochondroplasia. The company will also share data from studies of the investigational long-acting medicine BMN 333.

The data include encouraging new imaging results from a post-hoc analysis of a randomized, double-blind, placebo-controlled trial evaluating the effect of VOXZOGO on the growth of the foramen magnum and craniocervical junction in children with achondroplasia, including infants.

"These findings provide important new insight into the potential effect of VOXZOGO on growth-related dimensions of the foramen magnum and craniocervical junction, regions of the skull implicated in cervicomedullary compression and serious complications in children with achondroplasia," said Greg Friberg, M.D., Executive Vice President and Chief Research & Development Officer at BioMarin. "The results also underscore the potential VOXZOGO treatment may offer when started as early as possible, when it can have the greatest impact. Together with the additional data we're presenting on bone strength, limb alignment, and our emerging work in hypochondroplasia and next-generation medicines such as BMN 333, these findings reflect the depth and breadth of our commitment to understanding and improving outcomes for children living with skeletal dysplasias."

BioMarin recently submitted a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for VOXZOGO for the treatment of hypochondroplasia. The company is also on track with its 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.

Additional data to be presented at the meeting include research in hypophosphatasia, as well as results from the Phase 3 ENERGY 3 study of BMN 401 in children ages 1 to 12 years with ENPP1 deficiency.

Below are key presentations at ASBMR, with all times listed in Eastern Daylight Time:

C-Type Natriuretic Peptide (CNP) Agonist CNP38 Promotes Longitudinal Bone Growth by Growth Plate Expansion in the Hyp Mouse Model of XLH
Oral Presentation #1138
Monday, Oct. 12, 11:30 – 11:42 a.m.

BMN 333, a Long-Acting Vosoritide Investigational Product, Promotes Endochondral Bone Growth and Skeletal Microarchitecture in Juvenile Mice and Nonhuman Primates
Poster Presentation #092
Friday, Oct. 9, 5:30 – 7 p.m.
Sunday, Oct. 11, 2 – 3:30 p.m.

Examining the Effect of Vosoritide Treatment on Bone Strength in Children With Achondroplasia
Poster Presentation #486
Saturday, Oct. 10, 2 – 3:30 p.m.

Efficacy and Safety of BMN 401 in Children With ENPP1 Deficiency: 52-Week Results From the Pivotal ENERGY 3 Trial
Poster Presentation #554
Saturday, Oct. 10, 2 – 3:30 p.m.

Economic Burden of Hypophosphatasia: A Systematic Literature Review
Poster Presentation #473
Saturday, Oct. 10, 2 – 3:30 p.m.

Development of BMN 333, a Long-Acting Version of Vosoritide for FGFR3-Related Dwarfism and Other Skeletal Dysplasias
Poster Presentation #115
Sunday, Oct. 11, 2 – 3:30 p.m.

Comparison of Quantitative Computed Tomography Bone Mineral Density Measurements of Vosoritide-Treated Children With Achondroplasia to an Untreated Achondroplasia Reference Population
Poster Presentation #474
Sunday, Oct. 11, 2 – 3:30 p.m.

Vosoritide Increases Growth Velocity in Children With Hypochondroplasia: Phase 3 Trial Results
Poster Presentation #552
Sunday, Oct. 11, 2 – 3:30 p.m.

The Effect of Vosoritide on Growth of the Foramen Magnum and Craniocervical Junction in Children With Achondroplasia
Poster Presentation #483
Sunday, Oct. 11, 2 – 3:30 p.m.

Molecular Insights into Vosoritide's Role in Enhancing Bone Growth and Mineralization
Poster Presentation #362
Sunday, Oct. 11, 2 – 3:30 p.m.

Understanding the Impact of Hypophosphatasia (HPP) on Health-Related Quality-of-Life and Activities of Daily Living: A Patient-Based Conceptual Model
Poster Presentation #011
Sunday, Oct. 11, 2 – 3:30 p.m.

Prospective, Observational Study of a Cohort of Hypophosphatasia Patients' Phenotypes Over Time and the Utility of Digital Outcomes Measures in This Population
Poster Presentation #521
Sunday, Oct. 11, 2 – 3:30 p.m.

ENPP1 Inhibition Improves the Bone Phenotype, Mobility, Muscle Strength and Endurance in Later-Onset HPP Mice
Oral Presentation #1139
Monday, Oct. 12, 11:42 – 11:54 a.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 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. 

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 email [email protected]. For more information about VOXZOGO, please visit www.voxzogo.com. For additional information regarding this product, please contact BioMarin Medical Information at [email protected]. 

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 the American Society for Bone and Mineral Research 2026 Annual Meeting (ASBMR), including the potential impact and safety of VOXZOGO in children with achondroplasia and hypochondroplasia; BioMarin's plans and expectations for the development of VOXZOGO for children with hypochondroplasia, including plans for submissions to the European Medicines Agency (EMA) and other regional health authorities as well as 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 an anticipated launch in 2027; 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; BioMarin's plans and expectations for the development of BMN 333; and BioMarin's estimate regarding incidence and total addressable patient population (TAPP) with respect to the conditions targeted by BioMarin's product candidates and commercial products, including achondroplasia and 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 EMA, the European Commission and other regulatory authorities; 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^®, BioMarin RareConnections^® and VOXZOGO^® are registered trademarks of BioMarin Pharmaceutical Inc.

SOURCE BioMarin Pharmaceutical Inc.

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