Data from an investigator-led study showed treatment with VOXZOGO resulted in sustained improvements in growth over three years in children with hypochondroplasia
New Phase 1 results also presented for investigational BMN 333 support potential weekly dosing in children with achondroplasia
SAN RAFAEL, Calif., June 16, 2026 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced new data from studies of VOXZOGO® (vosoritide) in children with hypochondroplasia and the investigational medicine BMN 333 in achondroplasia were presented at ENDO 2026, the Endocrine Society Annual Meeting, in Chicago.
New data from a Phase 2, investigator-sponsored three-year extension study of VOXZOGO in 13 children with hypochondroplasia, led by Andrew Dauber, M.D. and investigators at Children's National Hospital, demonstrated sustained improvements in annualized growth velocity (AGV) and height standard deviation score (SDS), with a favorable safety profile. Mean height SDS improved by 0.72 SD over three years of treatment, while mean AGV increased from 4.27 cm/year at baseline to 7.24 cm/year at year one (p<0.001) and remained above baseline through years two and three.
BioMarin recently announced positive topline results from CANOPY-HCH-3, its registration-enabling Phase 3 pivotal study evaluating VOXZOGO in children with hypochondroplasia. These results will be included in the supplemental New Drug Application submission to the U.S. Food and Drug Administration planned for the third quarter of 2026.
"Building on the excellent Phase 3 results we recently announced, these new longer-term data further reinforce the potential of VOXZOGO to meaningfully improve growth outcomes for children with hypochondroplasia," said Greg Friberg, M.D., Executive Vice President and Chief Research & Development Officer at BioMarin. "Importantly, we continue to observe sustained growth improvements over time and a favorable safety profile, adding to the growing body of evidence supporting VOXZOGO for this potential new indication."
New BMN 333 Phase 1 Data Support Advancement Into Late-Stage Development
Additional data being presented at ENDO 2026 highlight the potential of investigational BMN 333, BioMarin's long-acting C-type natriuretic peptide (CNP) for achondroplasia. In a Phase 1 single-ascending dose study in healthy adults, BMN 333 demonstrated sustained systemic exposure associated with prolonged pharmacodynamic target engagement, supporting a weekly dosing schedule. The maximum examined dose of BMN 333 (500 μg/kg) increased exposure to free CNP by more than 13 times compared to another long-acting CNP agent, reflecting the potential of BMN 333 to become the new standard of care in achondroplasia. BMN 333 was well tolerated across all dose levels evaluated, with no dose-limiting toxicities or treatment-related serious adverse events.
In April, BioMarin began enrolling patients in the registration-enabling Phase 2/3 study of BMN 333. A data update from the dose-finding segment of this study is expected in 2027.
Below are key presentations for achondroplasia and hypochondroplasia at ENDO, with all times listed in Central Daylight Time:
Vosoritide Treatment in Children With Hypochondroplasia: Three-Year Results From a Phase 2 Extension Trial
Oral Presentation #ORF47-08
Monday, June 15, 3 - 3:15 p.m.
BMN333 Achieves High Sustained Released Vosoritide Exposure With Favorable Safety: Phase1 Results That Support Phase2/3 Trials in Achondroplasia
Poster Presentation #SUN-212
Sunday, June 14, 9 a.m. - 4 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.
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). To fulfill this post-marketing requirement, BioMarin intends to use the ongoing open-label extension studies compared to available natural history.
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 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: the data to be presented at ENDO 2026, the Endocrine Society Annual Meeting, including oral and poster presentations; BioMarin's plans and expectations for the development of VOXZOGO for children with hypochondroplasia, including safety profile and potential benefits and plans to submit a supplemental New Drug Application to the U.S. Food and Drug Administration (FDA) during the third quarter of 2026; BioMarin's plans and expectations for the development of BMN 333 for children with achondroplasia, including plans to provide a data update from the dose finding segment of the registration-enabling Phase 2/3 study of BMN 333 in 2027; the safety profile and potential benefits of BMN 333, including the potential to become the new standard of care in achondroplasia; 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, 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 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.
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