Casimersen

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Casimersen
Clinical data
Trade namesAmondys 45
Other namesSRP-4045
AHFS/Drugs.comFDA Professional Drug Information
License data
Routes of
administration		Intravenous
Drug classAntisense oligonucleotide
ATC code
  • None
Legal status
Legal status
Identifiers
CAS Number
  • 1422958-19-7
DrugBank
UNII
KEGG
Chemical and physical data
FormulaC268H424N124O95P22
Molar mass7584.536 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]

The most common side effects include upper respiratory tract infections, cough, fever, headache, joint pain and throat pain.[2]

Casimersen was approved for medical use in the United States in February 2021,[1][2] 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]

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][2]

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.[2] 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.[2]

The U.S. Food and Drug Administration (FDA) granted the application for casimersen fast track, priority review, and orphan drug designations.[2][5] The FDA granted the approval of Amondys 45 to Sarepta Therapeutics, Inc.[2]

References[]

  1. ^ Jump up to: a b c d e "Amondys 45- casimersen injection". DailyMed. Retrieved 1 March 2021.
  2. ^ Jump up to: 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. Public Domain This article incorporates text from this source, which is in the public domain.
  3. ^ "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.
  4. ^ 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. 1828. Clifton, N.J. pp. 31–55. doi:10.1007/978-1-4939-8651-4_2. ISBN 978-1-4939-8650-7. PMID 30171533.
  5. ^ "Casimersen Orphan Drug Designations and Approvals". U.S. Food and Drug Administration (FDA). 4 June 2019. Retrieved 25 February 2021.

External links[]

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