In a promising development for the treatment of rare diseases, Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a frontrunner in precision genetic medicine, announced that the U.S. Food and Drug Administration (FDA) has accepted and filed the company’s efficacy supplement to the Biologics License Application (BLA) for ELEVIDYS (delandistrogene moxeparvovec-rokl), marking a pivotal moment in the battle against […]
Sarepta Therapeutics’ SRP-9001 (delandistrogene moxeparvovec) has received the backing of the Cellular, Tissue and Gene Therapies Advisory Committee (CTGTAC) of the US Food and Drug Administration (FDA) for accelerated approval for Duchenne muscular dystrophy (DMD). The FDA committee voted 8 to 6 in favor of the investigational gene therapy for the treatment of ambulatory patients […]
Sarepta Therapeutics said that it plans to file a biologics license application (BLA) with the US Food and Drug Administration (FDA) to seek accelerated approval for SRP-9001 (delandistrogene moxeparvovec) for the treatment of ambulant individuals with Duchenne muscular dystrophy (DMD). The investigational DMD gene therapy is being co-developed with Roche under a $2.85bn deal signed […]