STAT+: FDA reviewers had string of concerns about Sarepta’s gene therapy for Duchenne, documents show
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.
