Sarepta Moves Closer To FDA Approval For Duchenne Muscular Dystrophy Drugs

The U.S. Food and Drug Administration (FDA) accepted on Tuesday Sarepta Therapeutics Inc.’s (NASDAQ: SRPT ) supplemental New Drug Applications seeking to convert the accelerated approvals of AMONDYS 45 (casimersen) and VYONDYS 53 (golodirsen) into traditional approvals for Duchenne muscular dystrophy (DMD). • Sarepta Therapeutics shares are climbing with conviction. What’s fueling SRPT momentum? The FDA approved VYONDYS 53 in 2019 and AMONDYS 45 in 2021. The agency set a Prescription Drug User Fee Act target action date of Feb. 28, 2027. The applications are supported by findings from the Phase 3 ESSENCE confirmatory study, along with published real-world evidence and the established safety profiles of both exon-skipping therapies. Louise Rodino-Klapac, president of research and development and technical operations at Sarepta, said that more than 1,800 patients worldwide h...