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BioMarin Announces Positive Phase 3 Pivotal Study Results for VOXZOGO® (vosoritide) in Children with Hypochondroplasia

$BMRNPress releaseMay 20, 2026, 4:20 PM ETRead the release

BioMarin announced positive results from the pivotal Phase 3 CANOPY-HCH-3 study for VOXZOGO in children with hypochondroplasia. The study met its primary endpoint, showing a statistically significant annualized growth velocity improvement of 2.33 cm/yr versus placebo (p<0.0001), along with significant gains in standing height, height Z-score, and arm span. Safety findings were consistent with the established profile in achondroplasia with no new signals.

Key figures

Drug Name
VOXZOGO (vosoritide)
Phase Of Trial
Phase 3
Study name
CANOPY-HCH-3
Patient count
80
Agv improvement
2.33 cm/yr
P value primary
<0.0001
Snda submission timing
Q3 2026

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