|Trade names||Amondys 45|
|Drug class||Antisense oligonucleotide|
|Main uses||Duchenne muscular dystrophy (DMD)|
|Side effects||Upper respiratory tract infections, cough, fever, headache, joint pain, throat pain|
|Typical dose||30 mg/kg q 1 wk|
|Chemical and physical data|
|Molar mass||7584.536 g·mol−1|
Casimersen, sold under the brand name Amondys 45, is a medication used to treat Duchenne muscular dystrophy (DMD). Specifically it is used in cases that have a mutation of the dystrophin gene that is amenable to exon 45 skipping. It is given by gradual injection into a vein.
Common side effects include upper respiratory tract infections, cough, fever, headache, joint pain, and throat pain. Other side effects may include kidney problems. It is an antisense oligonucleotide of phosphorodiamidate morpholino oligomer (PMO).
Casimersen was approved for medical use in the United States in 2021. It is not approved in either Europe or the United Kingdom. In the United States a year or treatment for a child who weights 10 kg is about 264,000 USD as of 2022.
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. In this group it increases dystrophin made by skeletal muscle; however, other effects remain unclear.
The usual dose is 30 mg/kg via IV infusion once a week.
Duchenne Muscular Dystrophy is an X-linked recessive disorder that results in the absence of a functional dystrophin protein. 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.
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. 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.
The U.S. Food and Drug Administration (FDA) granted the application for casimersen fast track, priority review, and orphan drug designations. The FDA granted the approval of Amondys 45 to Sarepta Therapeutics, Inc. 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.
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