Sarepta Therapeutics recebeu a designação Fast Track para SRP-9001 pela FDA
O SRP-9001 é uma terapia genica em investigação destinada a produção direcionada da proteína da microdistrofina. Trata-se de uma terapia para distrofia muscular de Duchenne. A designação Fast Track é um processo que acelera os estudos de medicações e terapias, assim como o processo de aprovação para doenças graves e que possam atender às necessidades médicas não atendidas.
Confira o texto divulgado pela Sarepta Therapeutics.
Sarepta Therapeutics Receives Fast Track Designation for SRP-9001 Micro-Dystrophin Gene Therapy for the Treatment of Duchenne Muscular Dystrophy
07/24/20 8:30 AM EDT
CAMBRIDGE, Mass., July 24, 2020 (GLOBE NEWSWIRE) — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today 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.
About SRP-9001 SRP-9001 is an investigational gene transfer therapy designed to deliver the micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein. Sarepta is responsible for global development and manufacturing for SRP-9001 and plans to commercialize SRP-9001 in the United States. 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 has exclusive rights to the micro-dystrophin gene therapy program initially developed at the Abigail Wexner Research Institute at Nationwide Children’s Hospital.
Texto completo: https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-receives-fast-track-designation-srp-9001