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Reviewers at the Food and Drug Administration concluded that Sarepta Therapeutics did not show that its gene therapy for Duchenne muscular dystrophy will likely benefit patients and left key safety concerns, according to briefing documents released Wednesday.

The FDA documents, released ahead of an advisory committee hearing Friday, found that Sarepta’s clinical studies “conducted to date do not provide unambiguous evidence” that the therapy, known as SRP-9001, is “likely beneficial for ambulatory patients” with Duchenne.


Additionally, the reviewers feared that administering a potentially “ineffective gene therapy” could both expose patients to safety risks — such as liver damage — associated with viral gene therapy, while also making those patients ineligible for other gene therapies in the future.

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