ADC Reviews

SRP-9001 Gene Therapy Will File For Licensing

gene transfer

Sarepta Therapeutics Announces Intent to Submit an Accelerated Approval Biologics License Application for its Gene Therapy SRP-9001 to Treat Duchenne Muscular Dystrophy


On July 29, Sarepta Therapeutics announced that it plans to submit a Biologics License Application (BLA) to the FDA for SRP-9001 (delandistrogene moxeparvovec) in ambulatory patients with Duchenne muscular dystrophy (DMD) this fall for accelerated approval.

SRP-9001 is an investigational gene transfer therapy designed to deliver SRP-9001 to muscle tissue, targeting the functional components that produce dystrophin. Sarepta is responsible for the global development and manufacturing of the SRP-9001. In December 2019, the company reached a cooperation with Roche to jointly develop SRP-9001, with a total transaction value of US$2.85 billion (US$750 million in cash + US$400 million in equity investment + US$1.7 billion in mileage).

In July 2020, SRP-9001 received FDA Fast Track designation. Later, SRP-9001 also received Rare Pediatric Disease (RPD) designation in the U.S. and Orphan Drug designation in the U.S., EU, Switzerland and Japan.

Duchenne muscular dystrophy (DMD) is a rare and fatal neuromuscular genetic disorder. Globally, it affects 1 in every 3,500-5,000 male newborns. DMD is caused by changes or mutations in genes encoding dystrophin instructions. It usually occurs before the age of 5 years. The clinical feature is progressive symmetrical muscle weakness. It is more common in the proximal limbs, and the onset often begins in the lower limbs.