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Generic name: viltolarsen
Dosage form: injection, for intravenous use
Drug class: Miscellaneous uncategorized agents

Medically reviewed by Judith Stewart, BPharm. Last updated on Aug 24, 2023.

What is Viltepso?

Viltepso is used to treat Duchenne muscular dystrophy in adults and children who have a certain gene mutation. Your doctor will test you for this gene mutation.

Viltepso was approved by the US Food and Drug Administration (FDA) on an "accelerated" basis. In clinical studies, some people responded to Viltepso, but further studies are needed.


Tell each of your healthcare providers about all your medical conditions, allergies, and all medicines you use.

Before taking this medicine

Tell your doctor if you have ever had kidney problems.

Tell your doctor if you are pregnant or breastfeeding.

 How is Viltepso given?

Your doctor will perform blood tests to make sure you do not have conditions that would prevent you from safely using Viltepso.

Viltepso is given as an infusion into a vein, usually once per week. A healthcare provider will give you this injection.

Viltepso must be given slowly, and the infusion can take up to 60 minutes to complete.

Your kidney function may need to be checked while you are using this medicine.

What happens if I miss a dose?

Call your doctor for instructions if you miss an appointment for your treatment.

What should I avoid while receiving Viltepso?

Follow your doctor's instructions about any restrictions on food, beverages, or activity.

Viltepso side effects

Get emergency medical help if you have signs of an allergic reaction: hives, itching, rash, blistering or peeling; fever; difficult breathing; swelling of your face, lips, tongue, or throat.

Call your doctor at once if you have signs of kidney problems including:

Common side effects may include:

This is not a complete list of side effects and others may occur. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1 800-FDA-1088.

What other drugs will affect Viltepso?

Other drugs may affect Viltepso, including prescription and over-the-counter medicines, vitamins, and herbal products. Tell your doctor about all your current medicines and any medicine you start or stop using.

General information about the use of Viltepso

Popular FAQ

Viltepso's mechanism of action is to “hide” exon 53, causing cells to bypass this exon and permitting the remaining exons to fit together properly. This allows the body to form a shortened version of the dystrophin protein. Viltepso (viltolarsen) injection helps the body to make dystrophin, a protein found in muscle cells. Dystrophin helps to strengthen and protect muscles. When exons are missing, cells do not have the proper instructions to make dystrophin. Continue reading

The new drugs approved by the FDA for the treatment of Duchenne Muscular Dystrophy (DMD) are Amondys 45 (casimersen), Viltepso (viltolarsen), Vyondys 53 (golodirsen), Exondys 51 (eteplirsen), Elevidys (delandistrogene moxeparvovec), and Emflaza (deflazacort). They include antisense oligonucleotides, a glucocorticoid and a gene therapy.

Duchenne muscular dystrophy (DMD) is a genetic disease that causes progressive weakness, loss of motor function and damage to skeletal and heart muscles. DMD, which has no cure, primarily affects males starting in early childhood, usually between 2 and 3 years of age. Continue reading

The length of time it takes for Viltepso (viltolarsen) to work in Duchenne muscular dystrophy (DMD) is still being evaluated in an ongoing study. Small studies showed an increase in the dystrophin protein (a "marker") in the muscle cells of patients with DMD after 20 to 24 weeks. Researchers believe that this increase in dystrophin may predict a clinical benefit in patients, but that is not yet proven. Continue reading

Further information

Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.