Treats Duchenne muscular dystrophy (DMD). NDA 212154, held by Nippon Shinyaku. Viltolarsen.
As of Nov 14, 2026, NDA 212154 (Viltepso), held by Nippon Shinyaku, was first approved by FDA on Aug 12, 2020, with 1 approved supplement and 0 published complete response letters.
VILTEPSO 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 VILTEPSO. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.
| Date | Submission | Status | Documents |
|---|---|---|---|
| Aug 12, 2020 | Original applicationNew molecular entity · Priority review | Approved |
| Product | Active ingredient | Marketing |
|---|---|---|
| Viltepso250MG/5ML (50MG/ML) · solution;intravenous | Viltolarsen | RxReference drug |
| Mar 11, 2021 | Supplement 2Labeling · Standard review | Approved | |
| Aug 12, 2020 | 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
