BioMarin's VOXZOGO Shows Positive Phase 3 Results for Hypochondroplasia, Seeks FDA Expansion

BioMarin Pharmaceutical announced positive Phase 3 trial results for VOXZOGO in children with hypochondroplasia, a rare genetic bone growth disorder. The findings were published in NEJM Evidence. The company has simultaneously filed a supplemental FDA application to expand VOXZOGO's label, aiming for it to be the first approved targeted therapy for this condition, building on its existing approval for achondroplasia.