Treats Duchenne muscular dystrophy (DMD). NDA 211970, held by Sarepta Theraps Inc. Golodirsen.
As of Nov 14, 2026, NDA 211970 (Vyondys 53), held by Sarepta Theraps Inc, was first approved by FDA on Dec 12, 2019, with 3 approved supplements and 1 published complete response letter.
VYONDYS 53 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 53 skipping. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with VYONDYS 53. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials.
| Date | Submission | Status | Documents |
|---|---|---|---|
| Dec 12, 2019 | Original applicationNew molecular entity · Priority review | Approved |
| Letter | Product | Now |
|---|---|---|
| Aug 19, 2019 | Vyondys 53 (golodirsen)Clinical, Decision | Approved |
| Product | Active ingredient | Marketing |
|---|---|---|
| Vyondys 53100MG/2ML (50MG/ML) · solution;intravenous | Golodirsen | RxReference drug |
| Jun 4, 2024 | Supplement 10Labeling · Standard review | Approved | |
| Jun 4, 2024 | Supplement 11Labeling · Standard review | Approved | |
| Feb 11, 2021 | Supplement 2Labeling · Standard review | Approved | |
| Dec 12, 2019 | 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
