Viltepso (Viltolarsen) for Muscular dystrophy

Overview
Viltepso is approved by the U.S. Food and Drug Administration (FDA) under accelerated approval for treating Duchenne muscular dystrophy (DMD) in people with a specific mutation (variation) in the DMD gene that is responsive to exon 53 skipping. A person must have a confirmed genetic mutation before being eligible for treatment with Viltepso. Continued FDA approval may depend on further clinical trials showing significant patient benefit. Viltepso is also known by its drug name, viltolarsen.

Viltepso is an antisense oligonucleotide (a synthetic strand of DNA) that targets genetic material to help produce dystrophin, a protein crucial for muscle health. By increasing dystrophin levels, Viltepso may help slow the progression of DMD symptoms.

How do I take it?
Prescribing information states that Viltepso is administered through an intravenous (IV) infusion once weekly. The infusion typically lasts 60 minutes. Dose size is based on a person’s body weight. Depending on the volume, it may need to be diluted with 0.9 percent sodium chloride. This treatment should be administered exactly as prescribed by a health care provider.

Side effects
Common side effects of Viltepso include upper respiratory tract infections, injection site reactions, cough, and pyrexia (fever).

Rare but serious side effects may include kidney toxicity, so kidney function should be monitored closely during treatment. Standard creatinine tests may not effectively measure kidney health in people with DMD, so alternative kidney function tests are recommended.

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Label: Viltepso — Viltolarsen Injection, Solution — DailyMed