BioMarin's Voxzogo hits its goal in a second growth disorder

BioMarin Pharmaceutical said on 9 September 2026 that its growth drug Voxzogo (vosoritide) succeeded in a pivotal Phase 3 trial in children with hypochondroplasia — a genetic bone-growth condition that leaves children shorter than their peers. It is a potential second use for a drug already sold for achondroplasia, the most common form of dwarfism.

The CANOPY-HCH-3 study tested Voxzogo, a daily injection, against a placebo in 80 children aged 3 to 17. It met its main goal: children on the drug grew faster, gaining an extra 2.33 centimetres per year compared with placebo after 52 weeks — a result BioMarin said exceeded expectations and was highly statistically significant (p<0.0001). Children treated with Voxzogo also showed statistically significant increases in standing height and height Z-score versus placebo after one year.

The full dataset was published in NEJM Evidence and presented in a late-breaking oral session at the European Society for Paediatric Endocrinology (ESPE) 2026 Annual Meeting. BioMarin has submitted a supplemental New Drug Application to the U.S. Food and Drug Administration; if approved, Voxzogo would be the first targeted therapy for hypochondroplasia, with a potential 2027 launch.

Source: BioMarin, 9 September 2026.