Rare disease biotechnology company BioMarin Pharmaceutical Inc (Nasdaq:BMRN) announced on Wednesday that the Phase 3 CANOPY-HCH-3 study evaluating treatment with VOXZOGO (vosoritide) in children with hypochondroplasia met its primary endpoint, demonstrating a statistically significant increase in the change from baseline at week 52 in annualised growth velocity (AGV) compared to placebo.

Children who received VOXZOGO also showed a statistically significant increase in standing height and height Z-score versus placebo after one year of treatment, and BioMarin also reported significant improvements in arm span, a key prespecified secondary endpoint in the study.

These positive findings could have important implications for children with hypochondroplasia, offering improvements in reach, daily activities and independence, which have been highlighted as meaningful outcomes by the community, BioMarin said.

Safety findings in the study for VOXZOGO were consistent with the established profile in achondroplasia and no new safety signals were observed. Full results from the CANOPY-HCH-3 study will be presented at an upcoming medical meeting.

BioMarin plans to submit the supplemental New Drug Application (sNDA) to the US Food and Drug Administration (FDA) in the third quarter of 2026, followed by submissions to the European Medicines Agency (EMA) and other regional health authorities.