BioMarin Pharmaceutical Inc reported positive outcomes from its Phase 3 CANOPY-HCH-3 study of VOXZOGO (vosoritide) in children with hypochondroplasia, prompting a 4.8% rise in the company's shares in after-hours trading Wednesday.
The trial met its primary endpoint, registering a statistically significant improvement in annualized growth velocity of 2.33 cm per year relative to placebo at week 52. In its statement, the company said the results exceeded expectations.
Beyond the primary measure, the study recorded statistically significant increases in standing height and height Z-score. BioMarin also reported a statistically significant improvement in arm span at week 52 compared with placebo - a prespecified secondary endpoint. The company noted that gains in arm span could have practical consequences for children, potentially affecting reach, daily activities and independence.
Safety observations from the trial were described as consistent with the established safety profile for VOXZOGO in achondroplasia, with no new safety signals identified, according to the company.
BioMarin said it intends to file a supplemental New Drug Application with the U.S. Food and Drug Administration in the third quarter of 2026. The company added that submissions to the European Medicines Agency and other regional health authorities will follow. Full study data are scheduled to be presented at an upcoming medical meeting.
VOXZOGO is already approved for the related skeletal dysplasia achondroplasia. The CANOPY-HCH-3 results represent, according to the company, the first pivotal Phase 3 study in hypochondroplasia to demonstrate measurable gains in growth metrics.
Context and implications
The trial outcomes deliver multiple statistically significant growth measures at the one-year mark and set a clear regulatory pathway as signaled by the planned supplemental NDA filing in 2026. The company has indicated it will share the complete dataset at a forthcoming medical meeting, which will provide additional detail for clinicians and regulators.
Market participants reacted to the announcement with a notable after-hours uptick in the stock price, reflecting investor attention to clinical progress and the potential for an expanded label for VOXZOGO.