Exondys 51 (Eteplirsen) for Muscular dystrophy

Overview
Exondys 51 is approved by the U.S. Food and Drug Administration (FDA) for the treatment of Duchenne muscular dystrophy (DMD) in people with a confirmed mutation (variation) of the DMD gene that allows for exon 51 skipping. This approval is under the FDA’s accelerated approval pathway based on the increase in dystrophin (a muscle protein) observed in some people. Continued approval may depend on additional studies confirming clinical benefits. Exondys 51 is also known by its drug name, eteplirsen.

Eteplirsen is classified as an antisense oligonucleotide. It is thought to work by targeting specific genetic instructions within muscle cells, allowing for the production of dystrophin protein in individuals with certain mutations that affect the DMD gene. Increased dystrophin production may help slow the progression of DMD.

How do I take it?
Prescribing information states that Exondys 51 is administered as an intravenous infusion once weekly. The infusion is given over 35 to 60 minutes, and the solution must be diluted before administration. It is essential for this medication to be administered exactly as prescribed by a health care provider.

Side effects
Common side effects of Exondys 51 include balance disorder and vomiting.

Rare but serious side effects may include hypersensitivity reactions, such as bronchospasm (airway spasms), chest pain, cough, fast heart rate, and hives. If hypersensitivity occurs, medical providers may adjust the infusion rate or temporarily stop treatment.

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Label: Exondys 51 — Eteplirsen Injection — DailyMed