BioMarin获得Voxzogo第三阶段结果

2026-05-21 06:09

BioMarin Pharmaceutical (BMRN) is up ~3% in after-hours trading Wednesday after posting phase 3 data for Voxzogo (vosoritide) in children with hypochondroplasia that met its primary endpoint.
Children given the treatment saw a statistically significant increase in the change from baseline at week 52 in annualized growth velocity compared to placebo.
In addition, those children also saw significant increases in standing height and height Z-score versus placebo after one year.
Voxzogo was approved under accelerated approval in November 2021, meaning a post-market study is needed to confirm its efficacy and to allow it to remain on the market.
BioMarin said it would submit an sNDA to the US FDA to gain full approval in Q3.

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