Drugs / Casimersen
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) | AMONDYS 45 | 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 ↗ |
| Filed | US (FDA) | — | DMD-related muscular dystrophy | 2020-06-26 | “The Company has completed the submission of a rolling NDA to the U.S. Food and Drug Administration (FDA) seeking accelerated approval for casimersen (SRP-4045).” sarepta.com ↗ “submission of a rolling New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) seeking accelerated approval for casimersen (SRP-4045)” sarepta.com ↗ |
| Approved Indication not stated 1 milestone | |||||
| Approved | US (FDA) | AMONDYS 45 | — | 2021-02-25 | fda.gov ↗ |
Trials 4
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 11 trial | ||||||
| Phase 1 | NCT02530905 | Oct 2015 → Oct 2018 | Duchenne muscular dystrophy | Sarepta Therapeutics, Inc. | Completed | No outcome recorded |
| 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 |
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 |
| 2021-02-25 | Sarepta Therapeutics, Inc. | Regulatory Sarepta Therapeutics Announces FDA Approval of AMONDYS 45™ (casimersen) Injection for the Treatment of Duchenne Muscular Dystrophy (DMD) in Patients Amenable to Skipping Exon 45 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 AMONDYS 45 (casimersen). |
| 2020-08-25 | Sarepta Therapeutics, Inc. | Regulatory Sarepta Therapeutics Announces FDA Acceptance of Casimersen (SRP-4045) New Drug Application for Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 45 sarepta.com ↗ |
| 2020-08-05 | Sarepta Therapeutics, Inc. | Regulatory Sarepta Therapeutics Announces Second Quarter 2020 Financial Results and Recent Corporate Developments sarepta.com ↗
The Company has completed the submission of a rolling NDA to the U.S. Food and Drug Administration (FDA) seeking accelerated approval for casimersen (SRP-4045). |
| 2020-06-26 | Sarepta Therapeutics, Inc. | Regulatory Sarepta Therapeutics Completes Submission of New Drug Application Seeking Approval of Casimersen (SRP-4045) for Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 45 sarepta.com ↗ |
| 2019-03-28 | Sarepta Therapeutics, Inc. | Results Sarepta Therapeutics Announces Positive Expression Results from the Casimersen (SRP-4045) Arm of the ESSENCE Study sarepta.com ↗
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced results from its interim analysis of muscle biopsy endpoints comparing casimersen treatment to placebo in the ESSENCE study, also known as study 4045-301. |
All press releases naming this drug 10 releases
Evidence & citations 7 cited values
Every value below carries the sentence it was read from. 5 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | casimersen | “Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping will receive weekly IV infusions of casimersen at four escalating dose...” NCT02530905 ↗ |
| Known as | Amondys 45 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT04179409 ↗ |
| Known as | EXON-45: NG-12-0064 | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297566 ↗ |
| Known as | SRP-4045 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT02500381 ↗ |
| 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 | “SRP-4045 IV infusions” NCT02500381 ↗ |
| Target | DMD | “This drug is used to target skipping of exon 45 of the dystrophin gene.” NCT04179409 ↗ |