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.
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Exondys Approval: Measured Efficacy Outcomes Vs. Patient 'Anecdotes'
Pink Sheet’s Drug Review Profile looks at US FDA clinical memos opposing approval of Sarepta's Duchenne muscular dystrophy drug eteplirsen, revealing challenges patient advocates and agency face in transforming patient experiences into hard data that can be used for approvals.
Keeping Track: Ilaris Adds Three Rare Disease Claims; Sirukumab Submitted; Exondys 51's Surprising Approval
The latest drug development news and highlights from our FDA Performance Tracker.
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.