Positive vote from FDA Advisory Committee meeting for SRP 9001 gene therapy to treat Duchenne muscular dystrophy,- Sarepta Therapeutics.
Sarepta Therapeutics, Inc. announced that the FDA)Cellular, Tissue and Gene Therapies Advisory Committee (CTGTAC) voted 8 to 6 in support of accelerated approval of SRP 9001 (delandistrogene moxeparvovec) for the treatment of ambulatory patients with Duchenne muscular dystrophy with a confirmed mutation in the DMD gene.
“Today’s advisory committee outcome is extremely important to the patient community, who are in urgent need of new therapies,” said Doug Ingram, president and chief executive officer, Sarepta. “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.
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.
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