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Eteplirsen Review Offers Lessons For FDA, Advocacy Groups, Industry

 
• By Sue Sutter

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.

FDA, patient advocates and rare disease drug developers should take away several lessons from the development and regulatory review of Sarepta Therapeutics Inc.'s Duchenne muscular dystrophy drug Exondys 51 (eteplirsen).

FDA learned the challenges of integrating the patient voice in the regulatory decision-making process without its reviewers and staff

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