FDA Can’t Find Efficacy For BioMarin’s Drisapersen
This article was originally published in The Pink Sheet Daily
On verge of advisory committee meeting, FDA says development program was exemplary, but is disappointed data are inconclusive.
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Structured assessments, which are currently publicly available only for approved products, could help frame questions for panel discussion of specific drugs under review; FDA also exploring ways to make completed frameworks more readily accessible.
Circumstances of FDA's approval of Sarepta's Exondys 51 approval may give BioMarin reason to appeal the complete response for its Duchenne muscular dystrophy treatment drisapersen – but its high-quality data could preclude success.
Sarepta relied on the endpoint to get accelerated approval for eteplirsen, but the interim CEO says trials for follow-on products could use walk-run tests or other measures instead.