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Viltepso

viltolarsen

Used for Muscular Dystrophy

Viltepso (viltolarsen) is an injectable medication that's used for a rare, genetic condition called Duchenne Muscular Dystrophy (DMD). It was approved by the FDA through its Accelerated Approval Program. Viltepso (viltolarsen) is given as an intravenous (IV) infusion by a healthcare provider. Your child might need to receive this medication at an infusion center.

Reviewed by:

Ally Tsai, PharmD

Daisy Chau, PharmD

Last reviewed on April 28, 2022

Basics Side effects Pros & cons Pharmacist tips FAQs Warnings Dosage Alternatives Prices Medicare

What is Viltepso (viltolarsen)?

What is Viltepso (viltolarsen) used for?

Treatment of Duchenne Muscular Dystrophy (DMD) in people with a specific genetic mutation

How Viltepso (viltolarsen) works

Viltepso (viltolarsen) is an antisense oligonucleotide. It attaches to exon 53, a part of the gene that makes dystrophin. Dystrophin is a protein that keeps muscle cells intact and strong. In people with DMD, this protein is missing because of a mutation in exon 53. Viltepso (viltolarsen) works by exon skipping, meaning it cuts out exon 53 and its mutation from the finished protein. This helps to make a shorter but more functional dystrophin, which potentially provides strength to muscles and relief from symptoms of DMD.

Are you looking for information on Vyondys 53 (golodirsen) instead?

Drug Facts

Common BrandsViltepso

Drug ClassAntisense oligonucleotide

Controlled Substance ClassificationNot a controlled medication

Generic StatusNo lower-cost generic available

AvailabilityPrescription only

What are the side effects of Viltepso (viltolarsen)?

The following side effects may get better over time as your body gets used to the medication. Let your healthcare provider know immediately if you continue to experience these symptoms or if they worsen over time.

Common Side Effects

Upper respiratory tract infections (sneezing, runny, or stuffy nose; 63%)
Injection site reaction (redness, swelling; 25%)
Cough (19%)
Fever (19%)

Other Side Effects

Bruising
Hives
Joint pain
Diarrhea
Vomiting
Belly pain

Source: DailyMed

The following side effects have also been reported

Side effects that you should report to your care team as soon as possible:

Allergic reactions—skin rash, itching, hives, swelling of the face, lips, tongue, or throat
Kidney injury—decrease in the amount of urine, swelling of the ankles, hands, or feet

Side effects that usually do not require medical attention (report these to your care team if they continue or are bothersome):

Cough
Diarrhea
Fever
Pain, redness, or irritation at injection site
Runny or stuffy nose
Sore throat
Vomiting

Pros and cons of Viltepso (viltolarsen)

Pros

Can help some people with DMD who never had a treatment option before

Only needs to be injected once a week so it's convenient for parents with a busy schedule

Doesn't interact with other medications or supplements

Cons

Only available as a brand medication, so it might be expensive

More likely to cause severe kidney problems

Requires IV access, so it's not as convenient as taking a pill

Pharmacist tips for Viltepso (viltolarsen)

It takes about an hour to give the full dose of Viltepso (viltolarsen). Most commonly, your child will receive this medication at an infusion center or a provider's office. In some cases, it can be given at home by a home health nurse.

It's best to get your Viltepso (viltolarsen) injection around the same day every week. If you miss an infusion, talk with your provider right away about rescheduling your appointment.

If you have pain around the injection site after receiving Viltepso (viltolarsen), let your provider know. They can give you suggestions on how to ease the pain and discomfort from the needle.

Make sure your child gets routine blood and urine tests done on time while they are receiving Viltepso (viltolarsen). This is because this medication can cause kidney problems which can possibly be detected in these tests. By getting regular tests done, you and your child’s provider can continue to make sure this medication is safe for them.

Tell your provider if your child experiences any changes in weight while receiving Viltepso (viltolarsen) because the dose might need to be changed.

Frequently asked questions about Viltepso (viltolarsen)

What is Duchenne Muscular Dystrophy (DMD)?

DMD is a rare, genetic condition that causes muscle weakness and wasting in young people. Your body needs a specific protein called dystrophin to keep muscle cells intact. In people with DMD, this protein is either missing or present at lower levels. Over time, this can cause movement problems (e.g., repeated falls, difficulty walking), heart problems (e.g., irregular heartbeat, heart failure), and stomach problems (e.g., constipation, diarrhea). Viltepso (viltolarsen) helps to raise the levels of dystrophin in certain people, providing relief from symptoms of DMD.

Who can benefit from Viltepso (viltolarsen) the most?

Viltepso (viltolarsen) is used in certain people with DMD that have a mutated exon 53, a specific part of a gene. It skips (cuts out) exon 53 to help the body make a shortened dystrophin protein that somewhat works. About 8% of young people with DMD are affected by this mutation. Typically, a genetic test that looks at changes in DNA can help identify your child's specific mutation. There are also other "exon skipping" medications available to treat different mutations in exons, such as Exondys 51 (eteplirsen) and Amondys 45 (casimersen). If you have any questions about whether Viltepso (viltolarsen) is right for your child, follow up with your child's provider for more information.

What is the FDA’s Accelerated Approval Program?

The FDA's Accelerated Approval Program allows a new medication to be approved faster than usual if it fulfills an unmet need or if it treats serious or life-threatening conditions. These approvals are based on lab measurements that are thought to predict health outcomes before the medication shows clear benefits. For Viltepso (viltolarsen), the medication raised the amount of dystrophin in skeletal muscles from 0.6% to nearly 6%, which might predict better muscle control in certain people with DMD. After accelerated approval, the manufacturer is required to complete clinical studies to confirm beneficial health outcomes (in this case, if Viltepso (viltolarsen) improves the time to stand in people with DMD). Depending on the results of these studies, the FDA can either give the medication full approval or pull it off the market.

Is Viltepso (viltolarsen) a cure for DMD?

No, Viltepso (viltolarsen) isn't a cure for DMD. Currently, all available treatments or medications for DMD can only relieve symptoms of this condition. As mentioned in the above FAQ, there are ongoing studies to see if Viltepso (viltolarsen) actually improves the time to stand in people with DMD. Other treatments, such as prednisone, are commonly given to help slow down muscle weakness and improve walking. Physical therapy and gentle exercises (swimming, stretching) are recommended along with medications to build muscle strength and prevent joint stiffening. If you have concerns about symptoms of DMD, talk with your child's provider to determine the best treatment plan.

If Viltepso (viltolarsen) is given through my child’s IV port, what should I watch out for?

Viltepso (viltolarsen) is given by IV infusion, which means the medication is delivered directly into your vein. Some people might have a small device called an IV port inserted under their skin for routine infusions. It's important to keep your child's port and the surrounding area clean and dry to prevent infections. Check with your provider if you have questions about how to take care of your child's IV port. Let your provider know right away if your child has any signs and symptoms of infection, such as fever, redness and swelling around the port, or pain in the arm closest to the port.

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What are the risks and warnings for Viltepso (viltolarsen)?

Viltepso (viltolarsen) can cause some serious health issues. This risk may be even higher for certain groups. If this worries you, talk to your doctor or pharmacist about other options.

Kidney problems

Risk factor: History of kidney problems

Studies showed that some animals that received Viltepso (viltolarsen) developed serious kidney problems. While animal studies aren’t always representative of what happens in humans, people who received medications similar to Viltepso (viltolarsen) have also reported to have kidney problems that can be life-threatening.

You’ll need to get blood and urine tests done before you start treatment and while you receive Viltepso (viltolarsen) so your provider can check your kidney health. Please also talk with your provider if you or your child has face swelling, is urinating less than usual, or has brown urine while receiving Viltepso (viltolarsen) injections. These can be signs and symptoms of severe kidney problems.

Viltepso (viltolarsen) dosage forms

Typical dosing for Viltepso (viltolarsen)

Viltepso (viltolarsen) is an intravenous (IV) infusion that is administered by a healthcare provider. The dose depends on your weight and your provider will determine the appropriate dose for you each time.

In general, the typical dose is 80 mg/kg once a week injected into a vein.

What are alternatives to Viltepso (viltolarsen)?

There are a number of medications that your doctor can prescribe in place of Viltepso (viltolarsen). Compare a few possible alternatives below.

Viltepso (viltolarsen)

Used for:

Treatment of Duchenne Muscular Dystrophy (DMD) in people with a specific genetic mutation

View Prices

Vyondys 53 (golodirsen)