Skip to Content

Viltolarsen

Generic Name: viltolarsen (VIL toe LAR sen)
Brand Name: Viltepso
Dosage Forms: intravenous solution (50 mg/mL)

Medically reviewed by Drugs.com on March 5, 2021. Written by Cerner Multum.

What is viltolarsen?

Viltolarsen 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.

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

Viltolarsen may also be used for purposes not listed in this medication guide.

Warnings

Before using viltolarsen tell your doctor about all your medical conditions or allergies, all medicines you use, and if you are pregnant or breastfeeding.

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 viltolarsen given?

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

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

viltolarsen must be given slowly, and the infusion can take about 60 minutes to complete.

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

Viltolarsen doses are based on weight. Your dose needs may change if you gain or lose weight.

What happens if I miss a dose?

Call your doctor for instructions if you miss an appointment for your viltolarsen injection.

What happens if I overdose?

Since viltolarsen is given by a healthcare professional in a medical setting, an overdose is unlikely to occur.

What should I avoid while receiving viltolarsen?

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

Viltolarsen 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:

  • pink, brown, or red urine;

  • foamy urine; or

  • swelling in your face, hands, feet, or stomach.

Common side effects may include:

  • redness, bruising, or swelling where an injection was given;

  • fever;

  • cough; or

  • cold symptoms such as stuffy nose, sneezing, sore throat.

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.

Viltolarsen dosing information

Usual Adult Dose for Muscular Dystrophy:

80 mg/kg via IV infusion once a week

Comments:
-This drug has been approved under an accelerated approval process based on demonstrated increased dystrophin production in skeletal muscle observed in treated patients; continued approval may be contingent upon verification and description of clinical benefit in a confirmatory trial.

Use: For the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping.

Usual Pediatric Dose for Muscular Dystrophy:

80 mg/kg via IV infusion once a week

Comments:
-This drug has been approved under an accelerated approval process based on demonstrated increased dystrophin production in skeletal muscle observed in treated patients; continued approval may be contingent upon verification and description of clinical benefit in a confirmatory trial.

Use: For the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping.

What other drugs will affect viltolarsen?

Other drugs may affect viltolarsen, 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.

Frequently asked questions

Further information

Remember, keep this and all other medicines out of the reach of children, never share your medicines with others, and use this medication only for the indication prescribed.

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