VOXZOGO (Vosoritide)

Oct. 9, 2026: BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) said it will present data from 13 studies at the ASBMR 2026 Annual Meeting in Boston, centered on VOXZOGO (vosoritide), BMN 333, BMN 401, and other skeletal-disease programs.

For VOXZOGO in achondroplasia, new post-hoc imaging data from a randomized, double-blind, placebo-controlled trial will assess effects on growth of the foramen magnum and craniocervical junction in children, including infants. Additional posters cover bone strength, quantitative CT bone mineral density versus an untreated reference population, and molecular effects on bone growth/mineralization.

For VOXZOGO in hypochondroplasia, BioMarin said an sNDA has been submitted to the FDA, with EMA and other regional submissions on track; if approved, launch is projected in 2027. ASBMR will also include Phase 3 trial results showing increased growth velocity in children with hypochondroplasia.

Regulatory catalyst: a separate sNDA for full approval of VOXZOGO in children with achondroplasia is under FDA review, with a PDUFA target action date of Feb. 28, 2027.

Other programs: BMN 333 (long-acting vosoritide) preclinical data in juvenile mice and nonhuman primates and a development update for FGFR3-related dwarfism/other skeletal dysplasias; BMN 401 52-week pivotal Phase 3 ENERGY 3 results in children ages 1-12 with ENPP1 deficiency; and hypophosphatasia/XLH-related research presentations.