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Sarepta wins approval for second exon-skipping DMD therapy after disputing FDA decision

December 13, 2019 12:23 AM UTC
Updated on Dec 13, 2019 at 1:25 AM UTC

FDA reversed an August decision that rebuffed Sarepta’s Vyondys 53 golodirsen, granting accelerated approval to the therapy to treat Duchenne muscular dystrophy in patients amenable to exon 53 skipping. The decision came as a surprise late Thursday, when the company revealed for the first time that it had appealed a complete response letter in which the agency flagged infection and toxicity concerns.

Shares of Sarepta Therapeutics Inc. (NASDAQ:SRPT) was up $29.59 (29%) to $130.06 during after-hours trading...

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