Sarepta Therapeutics Receives Fast Track Designation for DMD Gene Therapy
July 27, 2020
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.
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