Sarepta Therapeutics announces intent to submit an accelerated approval BLA for its gene therapy SRP 9001 to treat Duchenne muscular dystrophy.

“We are delighted to confirm that based on FDA feedback 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. We look forward to a collaborative review commencing this year and running through the first half of 2023,” said Doug Ingram, president and chief executive officer, Sarepta Therapeutics. “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.”

SRP 9001 was granted Fast Track designation in July 2020, an FDA process designed to facilitate the development and expedited review of drugs that treat serious conditions and fill unmet medical needs. In addition to Fast Track, SRP 9001 has also been granted Rare Pediatric Disease (RPD) designation in the United States, and Orphan Drug status in the United States, the European Union, Switzerland and Japan.