Sarepta's Eteplirsen Review Delay Emboldens Duchenne Community
This article was originally published in The Pink Sheet Daily
Is the lack of rejection cause for optimism, or will eteplirsen end up like BioMarin's drisapersen?
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Documents also show that Sarepta officials begged CDER director for approval on multiple occasions prior to the agency's September 2016 decision.
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.
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.