BioMarin presents Voxzogo skeletal dysplasia data at ASBMR meeting

BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) presented data from 13 studies at the American Society for Bone and Mineral Research 2026 Annual Meeting in Boston, including results from studies of Voxzogo (vosoritide) in children with achondroplasia and hypochondroplasia.

Key data included a post-hoc analysis from a randomized, double-blind, placebo-controlled trial evaluating Voxzogo's effect on the growth of the foramen magnum and craniocervical junction in children with achondroplasia, including infants. Those regions of the skull are associated with cervicomedullary compression and related complications in achondroplasia patients.

Additional presentations covered bone strength, limb alignment, and data on the investigational long-acting medicine BMN 333. Phase 3 results from the ENERGY 3 study of BMN 401 in children ages 1 to 12 with ENPP1 deficiency were also shared, along with research in hypophosphatasia.

BioMarin recently filed a supplemental New Drug Application with the U.S. Food and Drug Administration for Voxzogo for the treatment of hypochondroplasia, and said it is on track with submissions to the European Medicines Agency and other regional health authorities. If approved, BioMarin said a potential launch could occur in 2027. The company estimates approximately 14,000 children within its global footprint may be eligible for treatment. No medicines are currently approved by the FDA or EMA for hypochondroplasia.

Voxzogo is currently approved in the U.S., Japan, and Australia to increase linear growth in children of all ages with achondroplasia with open epiphyses. A separate supplemental application seeking full U.S. approval for achondroplasia, incorporating long-term safety and efficacy data, has an FDA Prescription Drug User Fee Act target action date of Feb. 28, 2027.

The information in this article is based on a press release statement from BioMarin.

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