|
Synonyms: Amondys 45® | SRP-4045 | SRP4045
casimersen is an approved drug (FDA (2021))
Compound class:
Nucleic acid
Comment: Casimersen is an antisense phosphorodiamidate morpholino oligomer (ASO) that is approved to treat certain patients with Duchenne muscular dystrophy (DMD). It promotes functional dystrophin synthesis in patients who have dystrophin gene variants that are amenable to exon 45 skipping, that is sufficient to restore some muscle function and slow disease progression.
We have been unable to resolve the full sequence provided in the INN record for casimersen to a SMILES or full IUPAC descriptor. |
| No information available. |
Summary of Clinical Use  |
| The FDA approved casimersen in February 2021. It is indicated as a treament for patients with Duchenne muscular dystrophy (DMD) who have dystrophin gene variants that are amenable to exon 45 skipping |
| Clinical Trials | |||||
| Clinical Trial ID | Title | Type | Source | Comment | References |
| NCT03532542 | An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy | Phase 3 Interventional | Sarepta Therapeutics, Inc. | ||
| NCT04179409 | A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of Casimersen, Eteplirsen and Golodirsen in Subjects With Duchenne Muscular Dystrophy Carrying Eligible DMD Duplications | Phase 2 Interventional | Nationwide Children's Hospital | ||
| NCT02500381 | Study of SRP-4045 and SRP-4053 in DMD Patients | Phase 3 Interventional | Sarepta Therapeutics, Inc. | ||
| NCT02530905 | Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) Patients | Phase 1 Interventional | Sarepta Therapeutics, Inc. | ||
