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Sarepta Announces FDA Acceptance of sNDAs for AMONDYS 45® and VYONDYS 53®

SRPT
  • Sarepta Therapeutics announced that the FDA has accepted the supplemental New Drug Applications (sNDAs) for AMONDYS 45 (casimersen) and VYONDYS 53 (golodirsen) for Duchenne muscular dystrophy.
  • The FDA assigned a Prescription Drug User Fee Act (PDUFA) target action date of February 28, 2027, for these submissions.
  • The sNDA filings seek to convert the accelerated approvals of AMONDYS 45 and VYONDYS 53 into traditional approvals.
  • The applications rely on data from the ESSENCE confirmatory study, substantial real-world evidence, and consistent safety profiles observed over 144 weeks.
  • More than 1,800 patients worldwide have been treated with Sarepta's exon-skipping therapies, showing preserved muscle function and slowed disease progression.
  • The Phase 3 ESSENCE study did not meet its primary endpoint, but post-hoc analyses indicated increased dystrophin expression and reduced decline in 4-step ascend at week 96.
  • Real-world evidence indicates VYONDYS 53 is associated with a 7.5-year delay in the need for nighttime ventilation, while AMONDYS 45 is linked to slowed lung function decline.
  • Across the portfolio, real-world data suggests multi-year survival benefits, delays in loss of ambulation of 3 to 4 years, reduced risk of low left ventricular ejection fraction, and fewer hospital visits.
  • Safety information highlights risks of hypersensitivity reactions, including anaphylaxis, for VYONDYS 53 and AMONDYS 45, as well as kidney toxicity monitoring requirements for both therapies.
  • Adverse reactions for VYONDYS 53 include headache, pyrexia, fall, abdominal pain, nasopharyngitis, cough, vomiting, and nausea.
  • Adverse reactions for AMONDYS 45 include upper respiratory infections, cough, pyrexia, headache, arthralgia, and oropharyngeal pain.
  • Sarepta's portfolio includes EXONDYS 51 (eteplirsen), AMONDYS 45 (casimersen), and VYONDYS 53 (golodirsen), which target specific DMD gene mutations amenable to exon 51, 45, or 53 skipping.