In a sudden about-face, the Food and Drug Administration will hold a meeting of outside experts to consider whether or not to approve Sarepta’s experimental gene therapy for Duchenne muscular dystrophy.
The announcement Thursday comes just weeks after the company said the FDA had told it an advisory panel meeting to review the treatment, called SRP-9001, would not be necessary. The decision to review Sarepta’s gene therapy without input from outside experts surprised some analysts and patient advocates.
The one-time therapy aims to permanently halt the progressive loss of muscle function in boys with Duchenne. Currently, patients with limited mutations that cause Duchenne can be treated with medicines that require weekly injections.
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