 | |
| Clinical data | |
|---|---|
| Trade names | Amondys 45 |
| Other names | SRP-4045 |
| AHFS/Drugs.com | Monograph |
| License data |
|
| Routes of administration | Intravenous |
| Drug class | Antisense oligonucleotide |
| ATC code | |
| Legal status | |
| Legal status | |
| Identifiers | |
| CAS Number | |
| DrugBank | |
| UNII | |
| KEGG | |
| Chemical and physical data | |
| Formula | C268H424N124O95P22 |
| Molar mass | 7584.536 g·mol−1 |
Casimersen, sold under the brand name Amondys 45, is an antisense oligonucleotide medication used for the treatment of Duchenne muscular dystrophy (DMD) in people who have a confirmed mutation of the dystrophin gene that is amenable to exon 45 skipping.[1][2][3][4] It is an antisense oligonucleotide of phosphorodiamidate morpholino oligomer (PMO).[1] Duchenne muscular dystrophy is a rare disease that primarily affects boys.[5] It is caused by low levels of a muscle protein called dystrophin.[5] The lack of dystrophin causes progressive muscle weakness and premature death.[5]
The most common side effects include upper respiratory tract infections, cough, fever, headache, joint pain and throat pain.[2][5]
Casimersen was approved for medical use in the United States in February 2021,[1][2][6] and it is the first FDA-approved targeted treatment for people who have a confirmed mutation of the DMD gene that is amenable to skipping exon 45.[2]
- ^ a b c d "Amondys 45- casimersen injection". DailyMed. Retrieved 1 March 2021.
- ^ a b c d e "FDA Approves Targeted Treatment for Rare Duchenne Muscular Dystrophy Mutation". U.S. Food and Drug Administration (FDA) (Press release). 25 February 2021. Retrieved 25 February 2021.
This article incorporates text from this source, which is in the public domain.
- ^ "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" (Press release). Sarepta Therapeutics. 25 February 2021. Retrieved 25 February 2021 – via GlobeNewswire.
- ^ Rodrigues M, Yokota T (2018). "An Overview of Recent Advances and Clinical Applications of Exon Skipping and Splice Modulation for Muscular Dystrophy and Various Genetic Diseases". Exon Skipping and Inclusion Therapies. Methods in Molecular Biology. Vol. 1828. Clifton, N.J. pp. 31–55. doi:10.1007/978-1-4939-8651-4_2. ISBN 978-1-4939-8650-7. PMID 30171533.
{{cite book}}: CS1 maint: location missing publisher (link) - ^ a b c d Cite error: The named reference
Drug Trial Snapshot: Amondys 45was invoked but never defined (see the help page). - ^ "Drug Approval Package: Amondys 45". U.S. Food and Drug Administration (FDA). 18 March 2021. Retrieved 13 September 2021.
