Sarepta Therapeutics Receives Fast Track Designation for DMD Gene Therapy

Rare Daily Staff

The U.S. Food and Drug Administration granted Fast Track designation to Sarepta Therapeutics’ SRP-9001 (AAVrh74.MHCK7.micro-dystrophin), its experimental gene transfer therapy for the rare muscle wasting disease Duchenne muscular dystrophy.

SRP-9001 is an experimental gene transfer therapy designed to deliver the micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein.

In December 2019, the company announced a licensing agreement granting Roche the exclusive right to launch and commercialize SRP-9001 outside the United States. Sarepta is responsible for global development and manufacturing for SRP-9001 and plans to commercialize SRP-9001 in the United States.

Safety and tolerability data at one year from four clinical trial participants who received SRP-9001 from its Study 101 were recently published in JAMA Neurology. A second study, Study 102, a randomized, double-blind, placebo-controlled study of SRP-9001, is ongoing with results expected in early 2021.

The Fast Track designation is a 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 designation. SRP-9001 was previously granted Orphan Drug status in the United States, the European Union, and Japan.