BioMarin’s Voxzogo meets endpoint in Phase 3 pediatric hypochondroplasia trial

BioMarin (BMRN) Pharmaceutical announced 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 annualized growth velocity vs. placebo. Children who received Voxzogo also showed a statistically significant increase in standing height and height Z-score vs. placebo after one year of treatment. Additionally, significant improvements in arm span were demonstrated, a key prespecified secondary endpoint in the study. The 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. The sNDA submission to the FDA is planned for Q3, followed by submissions to the European Medicines Agency and other regional health authorities. There are currently no medicines approved by the FDA or the EMA for the treatment of hypochondroplasia.