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16:07 EDT Sarepta confirms FDA panel vote in favor of DMD gene therapy - Sarepta Therapeutics announced that the Food and Drug Administration Cellular, Tissue and Gene Therapies Advisory Committee voted 8 to 6 in support of accelerated approval of SRP-9001 for the treatment of ambulatory patients with Duchenne muscular dystrophy with a confirmed mutation in the DMD gene. SRP-9001 is intended to treat the underlying cause of Duchenne, which is characterized by mutations in the dystrophin gene that results in the lack of dystrophin protein. In the absence of dystrophin, which is required to strengthen and protect muscles, muscles become weakened and damaged, the company said in a statement. "The committee's positive vote is based on the evaluation of the totality of evidence including the SRP-9001 product design as well as biological and empirical data," it added. The Biologics License Application for SRP-9001 is currently under priority review by the FDA with a regulatory action date of May 29. "With the May 29 action date our top priority, we will work collaboratively with the FDA to complete the review of our BLA for SRP 9001," said Doug Ingram, CEO of Sarepta.
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