Sarepta Shares Jump 6% as FDA Accepts sNDAs for Two DMD Drugs
SRPT•Sarepta’s supplemental NDAs for AMONDYS 45 and VYONDYS 53 seeking full FDA approval were accepted with a PDUFA target date of February 28, 2027. Shares climbed 6% after ESSENCE data showed positive trends in muscle-protecting protein and function retention, while Exondys 51 remains on accelerated approval without a full‐approval filing.
1. FDA Acceptance for AMONDYS 45 and VYONDYS 53
The FDA accepted Sarepta’s supplemental New Drug Applications to convert AMONDYS 45 (casimersen) and VYONDYS 53 (golodirsen) from accelerated to traditional approvals, setting a PDUFA target action date of February 28, 2027.
2. ESSENCE Study Data
The ESSENCE confirmatory trial did not meet its primary endpoint but showed consistent positive trends, including increased dystrophin production and slower loss of function over two years, with no new safety signals identified.
3. Market Reaction and Implications
Shares rose about 6% after the announcement as full approvals would remove confirmatory study requirements, enhance long-term prescribing certainty and potentially strengthen revenue forecasts for the two DMD therapies.
4. Exondys 51 Approval Status
Exondys 51 remains on accelerated approval and Sarepta has not yet submitted a full-approval application, meaning continued confirmatory studies are still required under current regulations.
