Sarepta crashes at an unexpected roadblock for its gene therapy

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In a surprise decision, the Food and Drug Administration said it would convene an advisory committee to discuss Sarepta Therapeutics,srpt) muscular dystrophy gene therapy, and SRPT stock fell on Friday.




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Just two weeks ago, Sarepta said the FDA would not ask a panel of independent experts to weigh in on the risks and benefits of gene therapy for Duchenne muscular dystrophy. But the agency changed its mind during a late-night meeting, Sarepta said in a news release Thursday.

Sarepta is asking for accelerated approval, meaning it still hasn’t completed two Phase 3 studies of the drug SRPT-9001. But it assumes that treatments based on elevated levels of a key protein in patients work. Outside experts will debate whether the protein indicates a benefit for patients.

“While we now see the potential for greater near-term stock volatility with upcoming regulatory events, we still see many upsides for SRPT-9001,” RBC analyst Brian Abraham said in a note to clients.

In premarket trading at today’s stock market, SRPT stock fell 1.7% near 120.20.

More to follow.

Follow Allison Gatlin on Twitter @IBD_AGatlin,

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