Biomarin Pharmaceutical Inc. Presents New Data on the Effect of Long-Term Treatment with VOXZOGO on Arm Span, Bone Health and Growth in Children with Achondroplasia

Published: 2026-05-02 16:50:29 ET BMRN

Published on 05/02/2026 at 04:50 pm EDT

BioMarin Pharmaceutical Inc. 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. 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. 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. 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. 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.

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.