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# Sarepta BLA for Duchenne Muscular Dystrophy
- URL: https://www.fdaweb.com/sarepta-bla-for-duchenne-muscular-dystrophy/
- Published: 2022-11-28T12:00:00.000Z
- Updated: 2026-09-14T18:07:40.000Z
- Author: David McFarland
- Tags: Biologics, #legacy-id-D5153267

FDA has accepted for review a Sarepta Therapeutics BLA that is seeking accelerated approval for SRP-9001 (delandistrogene moxeparvovec) for treating ambulant individuals with Duchenne muscular dystrophy (DMD). The priority review submission has been given a user fee review action date of 5/29/2023.

SRP-9001 is an investigational gene therapy being developed in partnership with Roche. It is designed to deliver therapy to muscle tissue for the targeted production of functional components of dystrophin, according to the company.

DMD is a rare, fatal neuromuscular genetic disease that occurs in about one in every 3,500-5,000 newborn males worldwide, Sarepta says. It is caused by a change or mutation in the gene that encodes instructions for dystrophin. Those with the disease eventually face increasing difficulty in breathing due to respiratory muscle dysfunction, and cardiac dysfunction can lead to heart failure, it says.