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).
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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 have received Sarepta’s exon-skipping therapies, with continued observations suggesting preservation of muscle function and slower disease progression.
ESSENCE Data and Real-World Evidence Support Applications
Sarepta said the ESSENCE study did not meet its primary endpoint, although treatment groups showed numerical advantages over placebo.
The company said additional post-hoc analyses addressing disease progression variability and the effects of the COVID-19 pandemic found increased dystrophin expression at week 96, and consistent reductions in four-step ascend decline across multiple analyses.
The therapies were also well tolerated through 144 weeks, with no new safety signals reported.
According to Sarepta, published real-world studies have linked VYONDYS 53 to a 7.5-year delay in the need for nighttime ventilation, while AMONDYS 45 has been associated with slower declines in lung function and delayed need for cough assist devices.
Across its phosphorodiamidate morpholino oligomer portfolio, the company also cited evidence suggesting benefits in survival, delayed loss of ambulation, improved cardiac outcomes, and fewer hospital visits.
SRPT Stock Price Activity: Sarepta Therapeutics shares were up 5.93% at $18.03 at the time of publication on Tuesday, according to Benzinga Pro data.
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