Application supported by extensive long-term safety and efficacy findings from three continuing studies that include adult height data plus further clinical results beyond growth such as body proportions and arm span measured during extended observation

FDA PDUFA target action date set for Feb. 28, 2027

BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that the U.S. Food and Drug Administration has accepted the supplemental New Drug Application for VOXZOGO (vosoritide) seeking full approval in children with achondroplasia. The agency assigned a Prescription Drug User Fee Act target action date of Feb. 28, 2027.

"This filing rests on the most comprehensive evidence collected for any therapy in achondroplasia and underscores BioMarin dedication to skeletal growth research," said Greg Friberg, M.D., Executive Vice President and Chief Research and Development Officer. "The results show clear gains in several skeletal measures beyond yearly growth velocity. Full approval would make VOXZOGO the first treatment to move from accelerated to traditional status using a complete data set that covers adult height and additional outcomes tracked over many years."

The submission draws on substantial long-term safety and efficacy information from three ongoing trials (111-205, 111-208 and 111-302). These studies produced clinically relevant improvements in growth together with gains in skeletal measures such as body proportions and arm span. The package represents the longest efficacy and safety record for any medicine evaluated in achondroplasia.

VOXZOGO obtained FDA accelerated approval in 2021, allowing quicker access based on markers considered reasonably likely to forecast clinical benefit. The current application aims to satisfy the post-marketing requirement to verify that benefit and secure full approval, backed by extended data from the three trials that show meaningful advances in growth and skeletal health for children with achondroplasia.

About Achondroplasia

Achondroplasia is the most frequent skeletal dysplasia causing disproportionate short stature. It features slowed endochondral ossification that produces uneven short stature and irregular bone structure in the limbs, spine, face and skull base. The condition stems from an alteration in the FGFR3 gene, which normally restrains bone growth.

Over 80 percent of affected children have parents of typical height and inherit the condition through a new gene change. The global rate is roughly one in 25,000 births. VOXZOGO is under study in children whose growth plates remain open, generally those younger than 18 years. About 25 percent of individuals with achondroplasia fall into this group.

About VOXZOGO (vosoritide)

In children with achondroplasia, endochondral bone formation is inhibited by a gain-of-function change in FGFR3. VOXZOGO, a C-type natriuretic peptide analog, enhances signaling downstream of FGFR3 to encourage bone growth.

VOXZOGO is the sole approved therapy that supports growth in children with achondroplasia from birth onward, and international guidelines advise starting treatment as soon as feasible. Approved in 2021, the medicine has reached more than 5,000 infants and children across more than 50 countries. Ongoing studies continue to assess effects on arm span, leg bowing, body proportions, spinal structure and quality-of-life measures.

VOXZOGO holds approval in the United States, Japan and Australia to boost linear growth in children of any age who have open growth plates. In the European Union it is indicated for achondroplasia in children four months and older whose growth plates remain open, confirmed by genetic testing. The U.S. indication rests on accelerated approval tied to improved annualized growth velocity, with continued approval dependent on verification of clinical benefit in further trials. BioMarin plans to meet this requirement through the ongoing open-label extension studies compared with available natural history data.

Patient Support for VOXZOGO

BioMarin support services deliver a smooth treatment path led by Clinical Coordinators who have provided hundreds of family trainings since launch. Resources include a caregiver mentorship program linking parents with peers and a U.S. clinician directory to help families locate experienced achondroplasia providers.

VOXZOGO U.S. Important Safety Information

What is VOXZOGO used for?

VOXZOGO is a prescription medicine indicated to increase linear growth in children with achondroplasia who have open growth plates.
Approval rests on accelerated review based on improved annualized growth velocity. Continued approval may require confirmation of clinical benefit in additional trials.

What is the most important safety information about VOXZOGO?

VOXZOGO can cause a temporary drop in blood pressure in some patients. To lower this risk and related symptoms such as dizziness, fatigue or nausea, patients should eat a meal and drink 8 to 10 ounces of fluid within one hour before each dose.

What are the most common side effects of VOXZOGO?

Common reactions include injection-site issues (redness, itching, swelling, bruising, rash, hives and pain), elevated blood alkaline phosphatase, vomiting, joint pain, lowered blood pressure and stomachache. These do not represent all possible effects. Patients should consult their healthcare provider for medical advice on side effects.

How is VOXZOGO taken?

VOXZOGO is given once daily as a subcutaneous injection by a trained caregiver. Caregivers must receive proper instruction before administering the medicine. The product comes with Instructions for Use that should be reviewed with each refill.
Administer at approximately the same time each day. A missed dose may be given within 12 hours; otherwise skip it and resume the regular schedule.
Dosing is weight-based and adjusted during routine visits. Treatment stops when the healthcare provider determines growth is no longer possible.

What should you tell the doctor before or during treatment?

Inform the physician of all medical conditions, including heart disease, use of blood-pressure medications, kidney impairment, pregnancy or breastfeeding plans.
Disclose all medicines, vitamins and supplements the patient takes.

Side effects may be reported to BioMarin at 1-866-906-6100 or to the FDA at www.fda.gov/medwatch or 1-800-FDA-1088. Please consult the full Prescribing Information and Patient Information for additional details.

About BioMarin

BioMarin is a global biotechnology company focused on rare genetic diseases. Founded in 1997 and headquartered in San Rafael, California, the firm has nine commercial therapies and an active pipeline. It applies genetic science to develop medicines that deliver meaningful patient impact. Additional information is available at www.biomarin.com.

Forward-Looking Statements

This release contains forward-looking statements regarding BioMarin business prospects, including the supplemental New Drug Application for VOXZOGO, the PDUFA target date, the safety profile and potential benefits beyond height, and continued development across skeletal conditions. Actual results may differ materially due to risks such as clinical trial outcomes, regulatory decisions and factors detailed in BioMarin filings with the Securities and Exchange Commission, including the Quarterly Report on Form 10-Q for the quarter ended March 31, 2026. Investors should not place undue reliance on these statements, which speak only as of the date of this release. BioMarin undertakes no obligation to update them.