BioMarin’s Voxzogo hits Phase III goal in hypochondroplasia

21 May 2026

US rare disease drugmaker BioMarin (Nasdaq: BMRN) said its growth disorder therapy Voxzogo (vosoritide) met the primary endpoint in a pivotal Phase III study in children with hypochondroplasia, potentially opening the way for the first approved treatment for the condition.

Top-line data from the CANOPY-HCH-3 trial showed children receiving Voxzogo achieved a 2.33 cm/year improvement in annualized growth velocity over placebo after 52 weeks of treatment, with statistical significance of p<0.0001.

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