Treats Duchenne muscular dystrophy (DMD). NDA 206488, held by Sarepta Theraps Inc. Eteplirsen.
As of Nov 14, 2026, NDA 206488 (Exondys 51), held by Sarepta Theraps Inc, was first approved by FDA on Sep 19, 2016, with 8 approved supplements and 0 published complete response letters.
EXONDYS 51 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. This indication is approved under accelerated approval based on an increase in dystrophin in skeletal muscle observed in some patients treated with EXONDYS 51. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials.
| Date | Submission | Status | Documents |
|---|---|---|---|
| Sep 19, 2016 | Original applicationNew molecular entity · Priority review | Approved |
| Exondys 51100MG/2ML (50MG/ML) · solution;intravenous | Eteplirsen | RxReference drug |
| Exondys 51500MG/10ML (50MG/ML) · solution;intravenous | Eteplirsen | RxReference drug |
| Dec 27, 2024 | Supplement 35Labeling · Standard review | Approved | |
| Jan 18, 2022 | Supplement 29Labeling · Standard review | Approved | |
| Jan 18, 2022 | Supplement 28Labeling · Standard review | Approved | |
| Jan 18, 2022 | Supplement 27Labeling · Standard review | Approved | |
| Jul 7, 2020 | Supplement 19Labeling · Standard review | Approved | |
| Oct 11, 2018 | Supplement 9Labeling · Standard review | Approved | |
| Feb 8, 2018 | Supplement 6Labeling · Standard review | Approved | |
| Feb 22, 2017 | Supplement 3Labeling · Standard review | Approved | None posted |
| Sep 19, 2016 | Original applicationNew molecular entity · Priority review | Approved |
Approvals, supplements, products and documents come from FDA's Drugs@FDA data files, refreshed daily; Drugs@FDA lists approved applications only. Complete response letters are FDA's published letters. Drugs@FDA and FDA complete response letters
