Duchenne Development: Six-Minute Walk Losing Favor?
Executive Summary
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.
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Eteplirsen Review Offers Lessons For FDA, Advocacy Groups, Industry
Agency reviewers believed the line between patient input and external intimidation had been crossed; early data from the Sarepta muscular dystrophy drug's flawed development program stoked patient community expectations and made regulatory review difficult.