– Accepted for review with target action date of February 28, 2027
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted for filing the supplemental New Drug Applications (sNDAs) for AMONDYS 45® (casimersen) and VYONDYS 53® (golodirsen) for the treatment of Duchenne muscular dystrophy (DMD). The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action data of February 28, 2027.
The sNDA submissions seek conversion of the accelerated approvals of AMONDYS 45 and VYONDYS 53 to traditional approvals. The applications are supported by data from the ESSENCE confirmatory study, as well as substantial published real-world evidence and the favorable and consistent safety profiles of both exon-skipping therapies.