Viltolarsen

Viltolarsen is a prescription medication used to treat Duchenne muscular dystrophy (DMD) in patients with a confirmed DMD gene mutation that is amenable to exon 53 skipping.

• Viltolarsen is available under the following different brand names: Viltepso

Common side effects of Viltolarsen include:

• upper respiratory tract infection
• injection site reactions (bruising, redness, swelling)
• cough
• fever
• bruising
• joint pain
• diarrhea
• vomiting
• abdominal pain
• ejection fraction decreased
• hives

Serious side effects of Viltolarsen include:

• hives
• itching
• rash
• blistering or peeling
• fever
• difficult breathing
• swelling of the face, lips, tongue, or throat
• pink, brown, or red urine
• foamy urine
• swelling in the face, hands, feet, or stomach

Rare side effects of Viltolarsen include:

• none 

Seek medical care or call 911 at once if you have the following serious side effects:

• Severe headache, confusion, slurred speech, arm or leg weakness, trouble walking, coordination loss, unsteady, very stiff muscles, high fever, profuse sweating, or tremors;
• Serious eye symptoms such as sudden vision loss, blurred vision, tunnel vision, eye pain or swelling, or seeing halos around lights;
• Serious heart symptoms include fast, irregular, or pounding heartbeats; fluttering in the chest; shortness of breath; sudden dizziness, lightheadedness, or passing out.

This is not a complete list of side effects and other serious side effects or health problems that may occur because of the use of this drug. Call your doctor for medical advice about serious side effects or adverse reactions. You may report side effects or health problems to FDA at 1-800-FDA-1088.

Adult and pediatric dosage

Injection, solution

• 250 mg/5mL (50 mg/mL) single-dose vial

Duchenne muscular dystrophy

Adult and pediatric dosage

• 80 mg/kg IV once a week

Dosage Considerations – Should be Given as Follows:

• See “Dosages”

If your medical doctor is using this medicine to treat your pain, your doctor or pharmacist may already be aware of any possible drug interactions and may be monitoring you for them. Do not start, stop, or change the dosage of any medicine before checking with your doctor, healthcare provider, or pharmacist first.

• Viltolarsen has no noted severe interactions with any other drugs
• Viltolarsen has no noted serious interactions with any other drugs
• Viltolarsen has no noted moderate interactions with any other drugs
• Viltolarsen has no noted minor interactions with any other drugs

This information does not contain all possible interactions or adverse effects. Visit the RxList Drug Interaction Checker for any drug interactions. Therefore, before using this product, tell your doctor or pharmacist about all the products you use. Keep a list of all your medications with you and share this information with your doctor and pharmacist. Check with your healthcare professional or doctor for additional medical advice, health questions, or concerns.

Contraindications

• None

Effects of drug abuse

• None

Short-Term Effects

• See “What Are Side Effects Associated with Using Viltolarsen?”

Long-Term Effects

• See “What Are Side Effects Associated with Using Viltolarsen?”

Cautions

• Kidney toxicity
  • Kidney toxicity observed in animals
  • Clinical experience with viltolarsen is limited, and kidney toxicity, including potentially fatal glomerulonephritis, has been observed after the administration of some antisense oligonucleotides
  • Monitor kidney function during treatment
• Only urine expected to be free of the excreted drug should be used for monitoring of urine protein; urine obtained on the day of infusion, before infusion, or urine obtained at least 48 hours after the most recent infusion, may be used; alternatively, use a laboratory test that does not use the reagent pyrogallol red, as this reagent has potential to cross-react with any drug that is excreted in urine and thus lead to a false positive result for urine protein
• Serum creatinine may not reliably measure kidney function in patients with DMD owing to reduced skeletal muscle mass
• Measure serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio before initiating
• Consider measuring GFR using an exogenous filtration marker before initiating
• During treatment, monitor urine dipstick every month and serum cystatin C and urine protein-to-creatinine ratio every 3 months
• If persistent increased serum cystatin C or proteinuria is detected, refer to a pediatric nephrologist for further evaluation

Pregnancy and Lactation

• There are no human or animal data available
• Lactation
  • There are no human or animal data to assess the effect on milk production