Sarepta Intents To Seek Accelerated Approval For Gene Therapy Candidate

Sarepta Therapeutics SRPT announced its intention to submit Biologics License Application (BLA) seeking accelerated approval for SRP-9001 to treat ambulant individuals with Duchenne muscular dystrophy.

SRP-9001 (delandistrogene moxeparvovec) is an investigational gene therapy for Duchenne being developed in partnership with Roche.

Doug Ingram, President and Chief executive officer, said, "We are delighted to confirm that based on the feedback we received following a thorough and in-depth review, we intend to submit a BLA for our SRP-9001 gene therapy to treat Duchenne muscular dystrophy this fall. Duchenne robs children daily and hourly of their muscle, stealing them bit by bit from their families and loved ones. Guided by rigorous science and productive regulatory discussions, our goal is to move with the urgency desperately needed by the patient community, and our upcoming BLA filing for SRP-9001 serves that goal.”

The company anticipates a collaborative review commencing this year and running through the first half of 2023.

Earlier, the U.S. Food and Drug Administration (FDA) has granted SRP-9001, the Fast Track designation, Rare Pediatric Disease (RPD) designation and Orphan Drug status in the United States.

Additionally, the Orphan Drug status was granted by the regulatory authorities in the European Union, Switzerland and Japan.

Price Action : Sarepta shares are trading around 9 percent at $93.27 on Friday at the time of publication.