Amondys 45 (Casimersen) for Muscular dystrophy

Overview
Amondys 45 is approved by the U.S. Food and Drug Administration (FDA) for treating Duchenne muscular dystrophy (DMD) in people with a confirmed mutation (variation) in the DMD gene that is responsive to exon 45 skipping. This approval was granted through an accelerated approval process based on observed increases in production of dystrophin (a muscle protein) in skeletal muscle. However, ongoing studies are required to confirm clinical benefits. Amondys 45 is also known by its drug name, casimersen.

Amondys 45 is an antisense oligonucleotide that targets a specific part of the DMD gene. It is thought to work by promoting exon 45 skipping, which helps cells produce a functional form of dystrophin, a protein essential for muscle strength and stability, which is typically absent in people with DMD.

How do I take it?
Prescribing information states that Amondys 45 is administered as an intravenous (IV) infusion once weekly, with a dose based on body weight. Each infusion takes 35 to 60 minutes and is given through an in-line filter after dilution. This drug should be administered exactly as prescribed by a health care professional.

Side effects
Common side effects of Amondys 45 include upper respiratory tract infection, cough, fever, headache, joint pain, and oropharyngeal (throat) pain.

Rare but serious side effects may include severe hypersensitivity reactions such as angioedema (swelling under the skin) and anaphylaxis (severe allergic reaction). Amondys 45 may also carry a risk of kidney toxicity, and individuals should undergo regular kidney function monitoring.

For more information about this treatment, visit:

Amondys 45 Injection — DailyMed