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FDA grants accelerated approval to controversial muscular dystrophy drug

The Washington Post: After months of advocacy and speculation, the Food and Drug Administration today granted accelerated approval to the first treatment for a rare form of muscular dystrophy. The decision pitted the passionate testimony of patients and families against an FDA advisory committee and internal reviewers who weren't convinced the drug worked.

The approval of Sarepta Therapeutics' drug, eteplirsen, was a huge, emotionally fraught victory for families with Duchenne muscular dystrophy -- one of the most vocal and involved patient communities since the days when HIV patients pushed the agency to approve more drugs. The approval specifies that the clinical benefit of the drug "has not been established" and is contingent on a follow-up clinical trial. The process has been closely watched by parents, patient advocates and biotech investors, and has been seen as an important test case for the effort to integrate the patients’ point of view more deeply into the approval process.

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