Exondys 51
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.
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 Brands | Exondys 51 |
|---|---|
| Drug Class | Antisense oligonucleotide |
| Controlled Substance Classification | Not a controlled medication |
| Generic Status | No lower-cost generic available |
| Availability | Prescription only |
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%)
Less Common Side Effects
- Trouble breathing
- Blue lips
- Feeling weak
- Fever
- Flushing
- Dehydration
Exondys 51 (eteplirsen) 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
The following Exondys 51 (eteplirsen) 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.
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
Typical dosage 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.
