Viltolarsen

Viltolarsen is used to treat Duchenne muscular dystrophy in adults and children who have a specific gene mutation. Your doctor will check for this gene mutation.

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

Viltolarsen may also be used for reasons not mentioned in this medication guide.

Your doctor will do blood tests to ensure you do not have conditions that would make it unsafe for you to use viltolarsen.

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

viltolarsen must be given slowly, and the infusion may take around 60 minutes to finish.

Your kidney function might need to be monitored while you are using this medicine.

Viltolarsen doses are determined by your weight. Your dose may need to be adjusted if you gain or lose weight.

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

Tell your doctor if you have ever had kidney problems.

Tell your doctor if you are pregnant or breastfeeding.

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

Viltolarsen may cause serious side effects. Call your doctor immediately if you experience:

• pink, brown, or red urine;
• foamy urine; or
• swelling in your face, hands, feet, or stomach.

Common side effects of viltolarsen may include:

• redness, bruising, or swelling at the injection site;
• fever;
• cough; or
• cold symptoms such as a stuffy nose, sneezing, or sore throat.

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

Usual Adult Dose for Muscular Dystrophy:

80 mg/kg via IV infusion once a weekComments:-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 weekComments:-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.

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

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

Follow your doctor's instructions regarding any restrictions on food, drinks, or activities.

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.