Overview

Elevidys is approved by the U.S. Food and Drug Administration (FDA) for the treatment of Duchenne muscular dystrophy (DMD) in children aged 4 years and older who are ambulatory (still able to walk) and have a confirmed mutation (variation) in the DMD gene. Elevidys should not be used in people with preexisting liver impairment, recent infections or vaccinations, or with certain genetic deletions (specifically involving exon 8 and/or exon 9 of the DMD gene). Elevidys is also known by its drug name, delandistrogene moxeparvovec-rokl.

Elevidys is a gene therapy designed to deliver a shortened version of the dystrophin gene into muscle cells using a modified virus. This helps the body produce a functional form of dystrophin, a protein necessary for muscle strength and stability, which is missing or defective in people with DMD.

How do I take it?

Prescribing information states that Elevidys is given as a single intravenous (IV) infusion over one to two hours. Before treatment, a person must be screened for liver function, infections, and preexisting immunity to the treatment virus. Treatment typically requires beginning a corticosteroid regimen one day before infusion, which continues for at least 60 days. This treatment should be administered exactly as prescribed by a healthcare provider.

Side effects

Common side effects of Elevidys include vomiting, nausea, liver injury, fever, thrombocytopenia (low platelet counts), and elevated troponin-I (a marker of heart stress).

Rare but serious side effects may include serious infections, acute myocarditis (inflammation of the heart muscle), severe allergic reactions including anaphylaxis, and immune-mediated myositis (a condition where the immune system attacks muscle tissue). There is also a risk of acute liver failure, especially in people with existing liver problems.

For more information about this treatment, visit:

Elevidys (Delandistrogene Moxeparvovec-Rokl) Suspension, for Intravenous Infusion — Sarepta Therapeutics