Drugs / Golodirsen
Regulatory milestones approvals, filings & regulatory actions · 3 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed DMD-related muscular dystrophy 2 milestones | |||||
| Filed | US (FDA) | VYONDYS 53 | DMD-related muscular dystrophy | 2026-06-30 | “FDA has accepted for filing the supplemental New Drug Applications (sNDAs) for AMONDYS 45® (casimersen) and VYONDYS 53® (golodirsen)” sarepta.com ↗ “In April 2026, Sarepta submitted sNDAs seeking to convert AMONDYS 45 and VYONDYS 53 from accelerated to traditional approvals.” sarepta.com ↗ |
| Filed | US (FDA) | — | DMD-related muscular dystrophy | 2018-12-20 | “announced today that it has completed the submission of its rolling New Drug Application (NDA) seeking accelerated approval for golodirsen (SRP-4053)” sarepta.com ↗ “Filed NDA for golodirsen with priority review, PDUFA August 19th” sarepta.com ↗ |
| Approved Indication not stated 1 milestone | |||||
| Approved | US (FDA) | VYONDYS 53 | — | 2019-12-12 | fda.gov ↗ |
Trials 5
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT02310906 | Jan 2015 → Mar 2019 | Duchenne muscular dystrophy | Sarepta Therapeutics, Inc. | Completed | Mixed |
| Phase 21 trial | ||||||
| Phase 2 | NCT04179409 | Feb 2020 → Sep 2021 | Duchenne muscular dystrophy | Kevin Flanigan | Completed | No outcome recorded |
| Phase 32 trials · 1 missed | ||||||
| Phase 3 | NCT03532542 | Aug 2018 → Jul 2023 | Duchenne muscular dystrophy | Sarepta Therapeutics, Inc. | Terminated | No outcome recorded |
| Phase 3 | NCT02500381 | Sep 2016 → Nov 2024 | Duchenne muscular dystrophy | Sarepta Therapeutics, Inc. | Completed | Missed primary |
| Phase 41 trial | ||||||
| Phase 4 | NCT04708314 | Oct 2020 → May 2021 | Duchenne muscular dystrophy | Rare Disease Research, LLC | Terminated | No outcome recorded |
News releases announcing trial results or a regulatory action · 8
| Date | Issuer | Release |
|---|---|---|
| 2026-06-30 | Sarepta Therapeutics, Inc. | Regulatory Sarepta Announces FDA Acceptance of sNDAs for AMONDYS 45® and VYONDYS 53® sarepta.com ↗ |
| 2026-05-06 | Sarepta Therapeutics, Inc. | Regulatory Sarepta Therapeutics Announces First Quarter 2026 Financial Results and Recent Corporate Developments sarepta.com ↗
Completed submission of sNDA for AMONDYS 45 and VYONDYS 53 to FDA seeking conversion to traditional approval |
| 2025-11-03 | Sarepta Therapeutics, Inc. | Results Sarepta Therapeutics Announces Third Quarter 2025 Financial Results and Recent Corporate Developments, Including Completion of Its Confirmatory Study, ESSENCE sarepta.com ↗
While the ESSENCE study did not achieve statistical significance on its primary endpoint, results indicate positive and encouraging trends favoring therapy at 96 weeks |
| 2019-12-12 | Sarepta Therapeutics, Inc. | Regulatory Sarepta Therapeutics Announces FDA Approval of VYONDYS 53™ (golodirsen) Injection for the Treatment of Duchenne Muscular Dystrophy (DMD) in Patients Amenable to Skipping Exon 53 sarepta.com ↗
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 approved VYONDYS 53™ (golodirsen). |
| 2019-08-19 | Sarepta Therapeutics, Inc. | Regulatory Sarepta Therapeutics Receives Complete Response Letter from the US Food and Drug Administration for Golodirsen New Drug Application sarepta.com ↗ |
| 2019-02-14 | Sarepta Therapeutics, Inc. | Regulatory Sarepta Announces FDA Acceptance of Golodirsen (SRP-4053) New Drug Application for Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 53 sarepta.com ↗ |
| 2018-12-20 | Sarepta Therapeutics, Inc. | Regulatory Sarepta Therapeutics Completes Submission of New Drug Application Seeking Approval of Golodirsen (SRP-4053) in Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 53 sarepta.com ↗
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a leader in precision genetic medicine for rare diseases, announced today that it has completed the submission of its rolling New Drug Application (NDA) seeking accelerated approval for golodirsen (SRP-4053), a phosphordiamidate morpholino oligimer engineered to treat those patients with Duchenne muscular dystrophy who have genetic mutations subject to skipping exon 53 of the Duchenne gene. |
| 2017-09-06 | Sarepta Therapeutics, Inc. | Results Sarepta Therapeutics Announces Positive Results in Its Study Evaluating Gene Expression, Dystrophin Production, and Dystrophin Localization in Patients with Duchenne Muscular Dystrophy (DMD) Amenable to Skipping Exon 53 Treated with Golodirsen (SRP-4053) sarepta.com ↗ |
All press releases naming this drug 12 releases
Evidence & citations 7 cited values
Every value below carries the sentence it was read from. 6 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | golodirsen | “SRP-4053 (golodirsen) solution for IV infusion.” NCT02310906 ↗ |
| Known as | NG-12-0163 | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297762 ↗ |
| Known as | SRP-4053 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT02500381 ↗ |
| Known as | Vyondys 53 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT04179409 ↗ |
| Action | Restore | “Skipping of a single copy of the duplicated exon is expected to result in a wild-type (WT) DMD transcript allowing the expression of a WT, full length dystrophin protein.” NCT04179409 ↗ |
| Route | Intravenous | “All eligible patients from Part 1, as well as new patients, will receive SRP-4053 (golodirsen) 30 mg/kg/week IV infusions, weekly, for up to 168 weeks.” NCT02310906 ↗ |
| Target | DMD | “This drug is used to target skipping of exon 53 of the dystrophin gene.” NCT04179409 ↗ |