Saturday, July 25, 2020
Sarepta Therapeutics, Inc., the leader in precision genetic medicine for rare diseases, announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to SRP-9001 (AAVrh74.MHCK7.micro-dystrophin). SRP-9001 is an investigational gene transfer therapy intended to deliver its micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein. Safety and tolerability data at one year from four clinical trial participants who received SRP-9001 in Study 101 were recently published in JAMA Neurology, and 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 (RPD) designation. SRP-9001 was previously granted Orphan Drug status in the United States, the European Union and Japan.