BioMarin's stock climbed 4.8% in after‑hours trading after its Phase 3 CANOPY‑HCH‑3 study met primary endpoint.
The trial showed a statistically significant 2.33 cm/yr increase in annualized growth velocity versus placebo at week 52.
Secondary endpoints also improved, with significant gains in standing height, height Z‑score, and arm span, without new safety signals.
BioMarin will file a supplemental NDA to the FDA in Q3 2026, then seek EMA and other regional approvals.