Bond.az - BioMarin Pharmaceutical Inc (NASDAQ:BMRN) shares rose 4.8% in after-hours trading Wednesday following positive results from its Phase 3 CANOPY-HCH-3 study evaluating VOXZOGO (vosoritide) in children with hypochondroplasia.
The study met its primary endpoint, demonstrating a statistically significant improvement in annualized growth velocity of 2.33 cm/yr compared to placebo at week 52. The company stated the results exceeded expectations. The study also showed statistically significant increases in standing height and height Z-score.
BioMarin reported that the study demonstrated statistically significant improvement in arm span at week 52 compared to placebo, a key prespecified secondary endpoint. The company said these improvements could have implications for reach, daily activities and independence in children with hypochondroplasia.
Safety findings were consistent with the established profile in achondroplasia, with no new safety signals identified, according to the company.
BioMarin plans to submit a supplemental New Drug Application to the U.S. Food and Drug Administration in the third quarter of 2026, followed by submissions to the European Medicines Agency and other regional health authorities. Full data from the study will be presented at an upcoming medical meeting.
VOXZOGO is currently approved for achondroplasia, a related skeletal dysplasia condition. The positive trial results represent the first pivotal Phase 3 study in hypochondroplasia to demonstrate gains in growth metrics.
