VOXZOGO shows long-term benefits in children with achondroplasia, improving growth and bone health.

BioMarin Pharmaceutical announced new research showing that VOXZOGO (vosoritide) treatment in children with achondroplasia leads to significant improvements in arm span, bone density, and proportional skeletal growth over long-term use. Children treated after age 5 showed notable height gains compared to untreated groups. Additional studies on hypochondroplasia also showed positive bone health effects, with pivotal Phase 3 trial results expected in early 2026. These findings reinforce VOXZOGO's role as the only approved treatment for achondroplasia from birth, with ongoing research supporting early and sustained treatment benefits.