FDA accepts Sarepta's applications to convert two DMD drugs to full approval

Sarepta Therapeutics (NASDAQ: SRPT) announced that the U.S. Food and Drug Administration has accepted for filing supplemental New Drug Applications for two of its Duchenne muscular dystrophy treatments, AMONDYS 45 (casimersen) and VYONDYS 53 (golodirsen), with a PDUFA target action date of February 28, 2027.

The applications seek to convert the accelerated approvals of both drugs to traditional approvals. The submissions are supported by data from the Phase 3 ESSENCE confirmatory study, real-world evidence, and safety data from both therapies.

In the ESSENCE study, the primary endpoint was not met, though numerical trends favored treatment. Post-hoc analyses showed that at week 96, treatment was associated with increased dystrophin expression and a reduction in 4-step ascend decline. Both therapies were well tolerated over 144 weeks with no new safety signals.

Real-world data cited in the applications include findings that VYONDYS 53 treatment is associated with a 7.5-year delay in the need for nighttime ventilation, and AMONDYS 45 treatment is associated with a statistically significant slowing of lung function decline.

"The submissions draw on the ESSENCE study and years of published real-world evidence, which together offer a fuller understanding of how these therapies benefit patients and change the progression of disease," said Louise Rodino-Klapac, Ph.D., president of research and development and technical operations at Sarepta.

Sarepta states that more than 1,800 patients worldwide have been treated across its exon-skipping therapies, which also include EXONDYS 51 (eteplirsen). AMONDYS 45 and VYONDYS 53 target patients with DMD mutations amenable to exon 45 and exon 53 skipping, respectively.

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