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Exondys 51

eteplirsen

Used for Muscular Dystrophy

Exondys 51 (eteplirsen) is an injectable medication that's used for a rare, genetic condition called Duchenne Muscular Dystrophy (DMD). It was the first medication approved for DMD through the FDA's Accelerated Approval Program. Exondys 51 (eteplirsen) is given as an intravenous (IV) infusion by a healthcare provider. Your child will need to take a genetic test before starting this medication to make sure it's right for them.

Reviewed by:

Ally Tsai, PharmD

Daisy Chau, PharmD

Last reviewed on April 28, 2022

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

What is Exondys 51 (eteplirsen)?

What is Exondys 51 (eteplirsen) used for?

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

How Exondys 51 (eteplirsen) works

Exondys 51 (eteplirsen) is an antisense oligonucleotide. It attaches to exon 51, 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 51. Exondys 51 (eteplirsen) works by exon skipping, meaning it cuts out exon 51 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.

Drug Facts

Common BrandsExondys 51

Drug ClassAntisense oligonucleotide

Controlled Substance ClassificationNot a controlled medication

Generic StatusNo lower-cost generic available

AvailabilityPrescription only

What are the side effects of Exondys 51 (eteplirsen)?

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

Problems with balance (38%)
Vomiting (38%)
Skin irritation (25%)
Headache (>10%)
Cough (>10%)
Rash (>10%)

Other Side Effects

Trouble breathing
Blue lips
Feeling weak
Fever
Flushing
Dehydration

Serious Side Effects

Contact your healthcare provider immediately if you experience any of the following.

Serious allergic reactions: trouble breathing, wheezing, chest pain, cough, fast heartbeat, hives

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

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

Cough
Headache
Loss of balance or coordination
Vomiting

Pros and cons of Exondys 51 (eteplirsen)

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 allergic reactions

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

Pharmacist tips for Exondys 51 (eteplirsen)

It takes about 35 minutes to 1 hour to give the full dose of Exondys 51 (eteplirsen). 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 Exondys 51 (eteplirsen) 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 Exondys 51 (eteplirsen), let your provider know. They might suggest using a topical cream on the area before injection to ease the pain and discomfort from the needle.

Tell your provider if your child experiences any changes in weight while receiving Exondys 51 (eteplirsen) because the dose might need to be changed.

Frequently asked questions about Exondys 51 (eteplirsen)

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). Exondys 51 (eteplirsen) helps to raise the levels of dystrophin in certain people, providing relief from symptoms of DMD.

Who can benefit from Exondys 51 (eteplirsen) the most?

Exondys 51 (eteplirsen) is used in certain people with DMD that have a mutated exon 51, a specific part of a gene. It skips (cuts out) exon 51 to help the body make a shortened dystrophin protein that somewhat works. About 13% 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 Vyondys 53 (golodirsen) and Amondys 45 (casimersen). If you have any questions about whether Exondys 51 (eteplirsen) 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 Exondys 51 (eteplirsen), the medication raised the amount of dystrophin in skeletal muscles, 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 Exondys 51 (eteplirsen) improves muscle control and other symptoms 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.

Can Exondys 51 (eteplirsen) improve my child's muscle control?

It's not currently clear whether Exondys 51 (eteplirsen) can improve muscle control in certain people with DMD. As mentioned in the FAQ above, it's only known that the medication raises the amount of dystrophin. More clinical studies are still required to make sure this medication can help with muscle movement. Typically, physical therapy and gentle exercises (e.g., swimming, stretching) are recommended along with Exondys 51 (eteplirsen) to build muscle strength and prevent joint stiffening. Talk with your provider to see what exercises can benefit your child while receiving this medication.

Can my child receive Exondys 51 (eteplirsen) if they have kidney problems?

Your child might be able to receive Exondys 51 (eteplirsen) if they have kidney problems. Keep in mind that studies showed that some animals that received this medication developed kidney problems. While animal studies don't always represent what happens in humans, it's important to look out for life-threatening kidney problems. Talk with your child's provider if your child is experiencing confusion, is urinating less than usual, or is swelling in the arms and legs while receiving Exondys 51 (eteplirsen) injections. These can be signs and symptoms of severe kidney problems.

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What are the risks and warnings for Exondys 51 (eteplirsen)?

Exondys 51 (eteplirsen) 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.

Allergic reaction

Exondys 51 (eteplirsen) can cause allergic reactions, which can be life-threatening. Watch out for signs and symptoms of allergic reactions, such as trouble breathing, wheezing, chest pain, cough, fast heartbeat, and hives. Let your provider know right away if allergic reactions occur. Your provider might slow the infusion or stop the treatment temporarily to help with your symptoms.

Exondys 51 (eteplirsen) dosage forms

Typical dosing for Exondys 51 (eteplirsen)

Exondys 51 (eteplirsen) 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 30 mg/kg once a week injected into a vein.

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