FDA advisory panel narrowly recommends approval for Sarepta’s gene therapy for Duchenne

For an archived version of a live blog about Friday’s FDA advisory panel hearing, click here.

A panel of independent advisers to the Food and Drug Administration narrowly voted 8-6 to recommend accelerated approval for Sarepta’s gene therapy for Duchenne muscular dystrophy, ruling that the potential benefit to patients outweighs a long list of concerns from the agency’s reviewers.

The close vote sets up a potential approval for a drug FDA reviewers had initially been planning to recommend rejecting, before a top official intervened to give the drug a public hearing. The FDA does not have to follow the recommendation of advisory panels but often does. It must make a decision by May 29.

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