Casimersen: Difference between revisions
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| legal_US_comment = <ref name="FDA PR 20210225" /><ref>https://www.amondys45.com/Amondys45_(casimersen)_Prescribing_Information.pdf</ref> |
| legal_US_comment = <ref name="FDA PR 20210225" /><ref>{{cite web | title = Amondys45 (casimersen) Prescribing Information | url = https://www.amondys45.com/Amondys45_(casimersen)_Prescribing_Information.pdf | work = Sarepta Therapeutics, Inc. }}</ref> |
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'''Casimersen''', sold under the brand name '''Amondys 45''', is |
'''Casimersen''', sold under the brand name '''Amondys 45''', is an [[Antisense RNA]] [[medication]] used to treat [[Duchenne muscular dystrophy]].<ref name="FDA PR 20210225">{{cite press release | title=FDA Approves Targeted Treatment for Rare Duchenne Muscular Dystrophy Mutation | website=U.S. [[Food and Drug Administration]] (FDA) | date=25 February 2021 | url=https://www.fda.gov/news-events/press-announcements/fda-approves-targeted-treatment-rare-duchenne-muscular-dystrophy-mutation-0 | access-date=25 February 2021}} {{PD-notice}}</ref><ref>{{cite press release | title=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 | publisher=Sarepta Therapeutics | via=GlobeNewswire | date=25 February 2021 | url=http://www.globenewswire.com/news-release/2021/02/25/2182845/0/en/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.html | access-date=25 February 2021}}</ref><ref name="Rodrigues_2018">{{cite journal | vauthors = Rodrigues M, Yokota T | title = An Overview of Recent Advances and Clinical Applications of Exon Skipping and Splice Modulation for Muscular Dystrophy and Various Genetic Diseases | journal = Methods in Molecular Biology | location = Clifton, N.J. | volume = 1828 | issue = | pages = 31–55 | date = 2018 | pmid = 30171533 | doi = 10.1007/978-1-4939-8651-4_2 }}</ref> |
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The most common side effects include upper respiratory tract infections, cough, fever, headache, joint pain and throat pain.<ref name="FDA PR 20210225" /> |
The most common side effects include upper respiratory tract infections, cough, fever, headache, joint pain and throat pain.<ref name="FDA PR 20210225" /> |
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Casimersen was approved for medical use in the United States in February 2021,<ref name="FDA PR 20210225" /> and it is the first FDA-approved targeted treatment for people who have a confirmed mutation of the DMD gene that is amenable to exon |
Casimersen was approved for medical use in the United States in February 2021,<ref name="FDA PR 20210225" /> and it is the first FDA-approved targeted treatment for people who have a confirmed mutation of the DMD gene that is amenable to [[exon skipping|skipping exon]] 45.<ref name="FDA PR 20210225" /> |
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== Medical uses == |
== Medical uses == |
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Revision as of 10:18, 28 February 2021
| Clinical data | |
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| Trade names | Amondys 45 |
| Other names | SRP-4045 |
| License data |
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| Routes of administration | Intravenous |
| Drug class | Antisense oligonucleotide |
| ATC code |
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| Legal status | |
| Legal status | |
| Identifiers | |
| CAS Number | |
| DrugBank | |
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Casimersen, sold under the brand name Amondys 45, is an Antisense RNA medication used to treat Duchenne muscular dystrophy.[1][3][4]
The most common side effects include upper respiratory tract infections, cough, fever, headache, joint pain and throat pain.[1]
Casimersen was approved for medical use in the United States in February 2021,[1] 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.[1]
Medical uses
Casimersen is indicated for the treatment of Duchenne muscular dystrophy (DMD) in people who have a confirmed mutation of the DMD gene that is amenable to exon 45 skipping.[1]
History
Casimersen was evaluated in a double-blind, placebo-controlled study in which 43 participants were randomized 2:1 to receive either intravenous casimersen or placebo.[1] All participants were male, between 7 and 20 years of age, and had a genetically confirmed mutation of the DMD gene that is amenable to exon 45 skipping.[1]
The U.S. Food and Drug Administration (FDA) granted the application for casimersen fast track, priority review, and orphan drug designations.[1][5] The FDA granted the approval of Amondys 45 to Sarepta Therapeutics, Inc.[1]
References
- ^ a b c d e f g h i j "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.
- ^ "Amondys45 (casimersen) Prescribing Information" (PDF). Sarepta Therapeutics, Inc.
- ^ "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". Methods in Molecular Biology. 1828. Clifton, N.J.: 31–55. doi:10.1007/978-1-4939-8651-4_2. PMID 30171533.
- ^ "Casimersen Orphan Drug Designations and Approvals". U.S. Food and Drug Administration (FDA). 4 June 2019. Retrieved 25 February 2021.
External links
- "Casimersen". Drug Information Portal. U.S. National Library of Medicine.
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