RARE Daily

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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