Eteplirsen
Generic name: eteplirsen [ e-TEP-lir-sen ]
Brand name: Exondys 51
Dosage form: intravenous solution (50 mg/mL)
Drug class: Miscellaneous uncategorized agents
What is eteplirsen?
Eteplirsen works by helping the body produce a protein called dystrophin. Dystrophin is a protein that helps stabilize the structure of muscle tissue and is necessary for proper muscle development and function. Without dystrophin, muscle fibers can become weak and easily damaged. This can affect voluntary movement such as walking.
Children with Duchenne muscular dystrophy have a mutation of a gene that would normally cause the body to produce dystrophin.
Eteplirsen is used in children with Duchenne muscular dystrophy (DMD). Eteplirsen is not a cure for DMD, but eteplirsen may lessen muscle weakness and muscle wasting caused by this disease.
Eteplirsen was approved by the US Food and Drug Administration (FDA) on an "accelerated" basis. In clinical studies, some children responded to this medicine. However, further studies are needed to determine if eteplirsen is generally effective in treating DMD.
Eteplirsen may also be used for purposes not listed in this medication guide.
Warnings
Before your child receives eteplirsen, tell the doctor about all your child's medical conditions or allergies, and all the medicines the child uses.
Before taking this medicine
Tell your child's doctor if any prior eteplirsen injections caused a severe allergic reaction in your child.
Before your child receives eteplirsen, tell the doctor about all your child's medical conditions or allergies, and all the medicines the child uses.
Although eteplirsen has been used mainly in children, it is not known whether eteplirsen could be harmful if used during pregnancy or while nursing.
How is eteplirsen given?
Eteplirsen is given as an infusion into a vein. A healthcare provider will give you this injection.
To make the injection more comfortable, a numbing medicine may be applied to the skin area where the IV needle will be placed.
Eteplirsen is given once per week. This medicine must be injected slowly, and the infusion can take up to 1 hour to complete.
Tell your caregivers if you feel itchy, warm, light-headed, or have chest tightness or trouble breathing when eteplirsen is injected.
Tell your doctor if your child has any changes in weight. Eteplirsen doses are based on weight, and any changes may affect the dose.
What happens if I miss a dose?
Call your child's doctor for instructions if you miss an appointment for your child's eteplirsen injection.
What happens if I overdose?
Seek emergency medical attention or call the Poison Help line at 1-800-222-1222.
What should I avoid while receiving eteplirsen?
Follow your doctor's instructions about any restrictions on food, beverages, or activity.
Eteplirsen side effects
Get emergency medical help if your child has signs of an allergic reaction: hives; difficult breathing; swelling of your face, lips, tongue, or throat.
Common side effects of eteplirsen may include:
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pain, bruising, itching, skin rash, or other irritation where the medicine was injected;
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joint pain;
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problems with balance; or
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cold symptoms such as stuffy nose, sneezing, sore throat.
This is not a complete list of side effects and others may occur. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.
Eteplirsen dosing information
Usual Adult Dose for Muscular Dystrophy:
30 mg/kg IV once a week as an IV infusion over 35 to 60 minutes
Comments:
-This drug is approved under accelerated approval based on an increase in dystrophin in skeletal muscle observed in some patients; a clinical benefit has not been established
-Continued approval may be contingent upon clinical benefit in confirmatory trials.
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 51 skipping
Usual Pediatric Dose for Muscular Dystrophy:
30 mg/kg IV once a week as an IV infusion over 35 to 60 minutes
Comments:
-This drug is approved under accelerated approval based on an increase in dystrophin in skeletal muscle observed in some patients; a clinical benefit has not been established
-Continued approval may be contingent upon clinical benefit in confirmatory trials.
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 51 skipping
What other drugs will affect eteplirsen?
Other drugs may affect eteplirsen, including prescription and over-the-counter medicines, vitamins, and herbal products. Tell each of your child's healthcare providers about all medicines the child uses now, and any medicine the child starts or stops using.
Popular FAQ
Exondys 51 is not technically gene therapy, but it does treat people with Duchenne muscular dystrophy who have a confirmed genetic mutation in their dystrophin gene which means they are unable to make the protein dystrophin. Dystrophin is needed for muscle repair. Without it, muscle cells become damaged and weakened over time, which can affect voluntary movement, such as walking. Continue reading
Exondys 51 can only be used by patients with DMD who have a confirmed mutation in their dystrophin gene that can be treated by skipping exon 51. This works out to be about 13% of people with DMD. Continue reading
There is currently no specific age range specified for using Exondys 51, but a two-part Phase 3 study scheduled to run until 2026 has recruited boys and young men aged 4 though 21 who meet the study criteria. Previous studies have only investigated boys up to age 19 years. Symptoms of Duchenne muscular dystrophy (DMD) usually appear between 3 and 5 years of age and the condition primarily affects boys at a rate of about 1 in every 3,600 male infants born. If you have an older child, the best thing you can do is talk to your doctor about Exondys 51 treatment. Continue reading
Exondys 51 is administered once a week as a 35 to 60-minute intravenous infusion by your healthcare provider. The correct dosage of Exondys 51 is drawn up and then diluted in an infusion bag containing 0.9% sodium chloride to make a total volume of 100-150 mL. The diluted solution is then administered into one of your veins via an in-line 0.2-micron filter.
Continue reading
The new drugs approved by the FDA for the treatment of Duchenne Muscular Dystrophy (DMD) are Amondys 45 (casimersen), Viltepso (viltolarsen), Vyondys 53 (golodirsen), Exondys 51 (eteplirsen), and Emflaza (deflazacort).
Duchenne muscular dystrophy (DMD) is a genetic disease that causes progressive weakness, loss of motor function and damage to skeletal and heart muscles. DMD, which has no cure, primarily affects males starting in early childhood, usually between 2 and 3 years of age. Continue reading
More about eteplirsen
- Side effects
- Dosage information
- During pregnancy
- Drug class: miscellaneous uncategorized agents
- En español
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Further information
Remember, keep this and all other medicines out of the reach of children, never share your medicines with others, and use this medication only for the indication prescribed.
Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.
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