Sarepta, FDA And The Dangers Of Strong Early Results
The small Duchenne patient community is already convinced eteplirsen works, but FDA doesn't seem to agree and the advisory committee didn't seem to offer a way out except through a controlled trial.
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Duchenne Development: Six-Minute Walk Losing Favor?
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.
Sarepta's Eteplirsen Approved After Contentious Internal FDA Debate
CDER Director Woodcock grants accelerated approval after FDA Commissioner Califf refuses to overturn her finding that the drug's effect on dystrophin is reasonably likely to predict clinical benefit in Duchenne muscular dystrophy.
FDA Neurology Clinical Team Leader Departure May Be Mountain Disguised As Molehill
Attention to the departure of neurology reviewer Ron Farkas from FDA has focused on what it means for Sarepta’s eteplirsen application, but there are larger regulatory environment issues that deserve more scrutiny.