Casimersen

Casimersen
Names
Trade namesAmondys 45
Other namesSRP-4045
Clinical data
Drug classAntisense oligonucleotide
Main usesDuchenne muscular dystrophy (DMD)[1]
Side effectsUpper respiratory tract infections, cough, fever, headache, joint pain, throat pain[1]
WHO AWaReUnlinkedWikibase error: ⧼unlinkedwikibase-error-statements-entity-not-set⧽
Routes of
use		Intravenous
Typical dose30 mg/kg q 1 wk[1]
External links
AHFS/Drugs.comMonograph
Legal
License data
Legal status
Chemical and physical data
FormulaC268H424N124O95P22
Molar mass7584.536 g·mol−1

Casimersen, sold under the brand name Amondys 45, is a medication used to treat Duchenne muscular dystrophy (DMD).[1] Specifically it is used in cases that have a mutation of the dystrophin gene that is amenable to exon 45 skipping.[1] It is given by gradual injection into a vein.[1]

Common side effects include upper respiratory tract infections, cough, fever, headache, joint pain, and throat pain.[1] Other side effects may include kidney problems.[1] It is an antisense oligonucleotide of phosphorodiamidate morpholino oligomer (PMO).[1]

Casimersen was approved for medical use in the United States in 2021.[1] It is not approved in either Europe or the United Kingdom.[3] In the United States a year or treatment for a child who weights 10 kg is about 264,000 USD as of 2022.[4]

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] In this group it increases dystrophin made by skeletal muscle; however, other effects remain unclear.[1]

Dosage

The usual dose is 30 mg/kg via IV infusion once a week.[1]

Side effects

Common side effects include: headache, fever, joint pain, cough and cold symptoms.[5] Side effects may include: pink, brown, or foamy urine or swelling of the hands and feet.[5]

Pharmacodynamics

Duchenne Muscular Dystrophy is an X-linked recessive disorder that results in the absence of a functional dystrophin protein.[6] Dystrophin protein is a protein that consists of an N-terminal actin-binding domain, C-terminal B-dystroglycan- binding domain, and 24 internal spectrum-like repeats. Dystrophin plays a role in muscle function and without dystrophin, muscle tissue will be replaced with fibrous and adipose tissue. Casimersen is an antisense phosphorodiamidate morpholino oligonucleotide designed to bind to the exon 45 of the DMD pre-MRNA, which prevents its exclusion into the mature RNA before translation. This change causes the production of an internally truncated dysphotrophin protein.[6]

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][7] The FDA granted the approval of Amondys 45 to Sarepta Therapeutics, Inc.[2] 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]

References

  1. 1 2 3 4 5 6 7 8 9 10 11 12 13 14 "Amondys 45- casimersen injection". DailyMed. Archived from the original on 3 August 2021. Retrieved 1 March 2021.
  2. 1 2 3 4 5 6 7 "FDA Approves Targeted Treatment for Rare Duchenne Muscular Dystrophy Mutation". U.S. Food and Drug Administration (FDA) (Press release). 25 February 2021. Archived from the original on 3 August 2021. Retrieved 25 February 2021. Public Domain This article incorporates text from this source, which is in the public domain.
  3. ↑ "Casimersen". SPS - Specialist Pharmacy Service. 16 March 2018. Archived from the original on 16 October 2021. Retrieved 31 October 2022.
  4. ↑ "Amondys 45 Prices, Coupons, Copay & Patient Assistance". Drugs.com. Archived from the original on 19 April 2021. Retrieved 31 October 2022.
  5. 1 2 "Casimersen Uses, Side Effects & Warnings". Drugs.com. Archived from the original on 13 May 2022. Retrieved 13 May 2022.
  6. 1 2 "Casimersen". go.drugbank.com. Archived from the original on 6 May 2022. Retrieved 6 May 2022.
  7. ↑ "Casimersen Orphan Drug Designations and Approvals". U.S. Food and Drug Administration (FDA). 4 June 2019. Archived from the original on 24 September 2021. Retrieved 25 February 2021.
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