BioMarin’s VOXZOGO Yields 13.6 cm Height Gain and Stable Bone Density
Extension trial data showed VOXZOGO treatment produced 10.6 cm gain at six years and 13.6 cm at eight years, with maintained bone mineral density. A hypochondroplasia study showed 12-month BMD increase of 0.03 g/cm2 and BMC increase of 54.8 g; pivotal Phase 3 toplines expected 1H2026.
1. Long-Term Achondroplasia Outcomes
Data from three ongoing long-term extension trials demonstrate that children with achondroplasia receiving VOXZOGO achieved proportional skeletal growth, with arm span Z-scores improving from baseline. Mean standing-height gains versus natural history cohorts reached 10.6 cm after six years and 13.6 cm after eight years, while annual DXA assessments confirmed maintained bone mineral density over time.
2. Hypochondroplasia Study and Phase 3 Outlook
In a single-arm Phase 2 trial involving 119 children with hypochondroplasia, 12-month treatment with VOXZOGO led to a 0.03 g/cm2 increase in bone mineral density and a 54.84 g rise in bone mineral content (p<0.0001). BioMarin plans to release topline results from its registration-enabling Phase 3 pivotal trial in the first half of 2026, targeting a submission for label expansion in the second half of the year.