With Voxzogo under pressure, BioMarin touts trial win in label expansion bid

BioMarin has announced positive Phase 3 pivotal study results for VOXZOGO (vosoritide) in children with hypochondroplasia, a related short‑stature condition, meeting its primary endpoint of significantly increasing annualized growth velocity compared to placebo (+2.33 cm/yr, p<0.0001).

VOXZOGO is already approved in the U.S., Japan, and Australia to increase linear growth in children of all ages with achondroplasia with open epiphyses, and is labeled in the EU for children 4 months and older; this latest win supports a potential label expansion into hypochondroplasia, a larger but less severe patient population.

The positive trial comes as VOXZOGO remains under FDA accelerated approval for achondroplasia, with BioMarin having submitted a U.S. sNDA in April 2026 seeking full approval, while also facing heightened competitive and regulatory pressure following recent safety reviews and trial setbacks in other indications such as Turner syndrome and SHOX deficiency.