Elevidys (Delandistrogene Moxeparvovec-Rokl) for Muscular dystrophy

Overview
Elevidys is a gene therapy approved by the U.S. Food and Drug Administration (FDA) for treating Duchenne muscular dystrophy (DMD) in people 4 years or older with a confirmed DMD gene mutation (variation). To be eligible, a person may be either ambulatory (able to walk) or nonambulatory (unable to walk). Approval for Elevidys for people who are nonambulatory is contingent upon further clinical studies that verify continued benefit. Elevidys is also known by its drug name, delandistrogene moxeparvovec-rokl.

This gene therapy works by delivering a modified gene to the recipient’s muscle cells, enabling them to produce a protein called micro-dystrophin. Scientists believe that micro-dystrophin, a shortened form of the muscle-supporting dystrophin protein, can help strengthen muscles and slow the progression of DMD.

How do I take it?
Prescribing information states that Elevidys is administered as a one-time intravenous (IV) infusion. Prior to infusion, a person should be screened for antibodies against the therapy’s viral vector. The recommended dose varies based on the recipient’s body weight. The drug is typically infused over one to two hours. The recipient typically starts a corticosteroid regimen one day before treatment to manage immune responses. This treatment should be administered exactly as prescribed by a health care provider.

Side effects
Common side effects of Elevidys include vomiting, nausea, liver injury, fever, and thrombocytopenia (low platelet counts).

Rare but serious side effects may include acute liver injury, myocarditis (inflammation of the heart muscle), immune-mediated myositis (severe muscle inflammation), and infusion-related reactions such as hypersensitivity and anaphylaxis. The recipient’s liver function and heart markers should be closely monitored to manage these risks.

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Label: Elevidys — Delandistrogene Moxeparvovec-Rokl Kit — DailyMed