PR Newswire·Sep 9, 2026, 9:05 AM ET

BioMarin Announces Positive Results of Phase 3 VOXZOGO® (vosoritide) Study in Hypochondroplasia Published in NEJM Evidence

BioMarin presented detailed Phase 3 CANOPY-HCH-3 data on VOXZOGO (vosoritide) for children with hypochondroplasia at the ESPE 2026 Annual Meeting & published results in NEJM Evidence.
The study demonstrated statistically significant improvements in annualized growth velocity (2.33 cm/year), standing height (2.35 cm), height Z-score (0.39), & arm span (1.03 cm) over 52 weeks compared to placebo.
BioMarin submitted a supplemental New Drug Application (sNDA) to the FDA seeking approval for VOXZOGO to treat hypochondroplasia, the first targeted therapy for this condition.
Safety findings were consistent with the established profile in achondroplasia, with most adverse events being mild & no treatment-related serious adverse events identified.
If approved, the treatment could launch in 2027, with parallel submissions underway for the European Medicines Agency & other regional authorities.
Hypochondroplasia is a rare genetic skeletal dysplasia affecting bone growth, with BioMarin estimating approximately 14,000 eligible children globally.
Currently, no medicines are approved by the FDA or EMA for the treatment of hypochondroplasia.
VOXZOGO is already approved for achondroplasia in the U.S., Japan, Australia, & the EU, having helped over 5,000 children in more than 50 countries since its 2021 approval.