What Is Exondys 51 Used For and How Does it Work?
Eteplirsen is used to treat Duchenne muscular dystrophy.
Eteplirsen is available under the following different brand names: Exondys 51.
What Are the Dosages of Exondys 51?
Dosages of Exondys 51:
Adult and Pediatric Dosage Forms and Strengths
IV Solution
- 50 mg/mL (as 100mg/2mL or 500mg/10mL single-dose vials)
Dosage Considerations – Should be Given as Follows:
Duchenne Muscular Dystrophy
- Indicated for Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping
- 30 mg/kg intravenously (IV) once weekly
Dosing Considerations
- Approved under accelerated approval based on an increase in dystrophin in skeletal muscle observed in some patients
- Clinical benefit has not been established
- Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials
What Are Side Effects Associated with Using Exondys 51?
Common side effects of Exondys 51 include:
- Balance disorder
- Vomiting
- Contact dermatitis
- Confusion
Other side effects of eteplirsen include:
- Transient redness
- Facial flushing
- The elevated temperature on days of infusion
This document does not contain all possible side effects and others may occur. Check with your physician for additional information about side effects.
What Other Drugs Interact with Exondys 51?
If your doctor has directed you to use this medication, 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, health care provider or pharmacist first.
EEteplirsen has no listed severe interactions with other drugs.
Eteplirsen has no listed serious interactions with other drugs.
Eteplirsen has no listed moderate interactions with other drugs.
Eteplirsen has no listed mild interactions with other drugs.
This document does not contain all possible interactions. Therefore, before using this product, tell your doctor or pharmacist of all the products you use. Keep a list of all your medications with you, and share the list with your doctor and pharmacist. Check with your physician if you have health questions or concerns.
What Are Warnings and Precautions for Exondys 51?
Warnings
This medication contains eteplirsen. Do not take Exondys 51 if you are allergic to eteplirsen or any ingredients contained in this drug.
Keep out of reach of children. In case of overdose, get medical help or contact a Poison Control Center immediately.
Contraindications
- None
Effects of Drug Abuse
- No information is available.
Short-Term Effects
- See "What Are Side Effects Associated with Using Exondys 51?"
Long-Term Effects
- See "What Are Side Effects Associated with Using Exondys 51?"
Cautions
- Approved under accelerated approval; clinical benefit has not been established
Pregnancy and Lactation
There are no human data available on eteplirsen to assess the use during pregnancy. Consult your doctor.
It is unknown if eteplirsen is distributed in human breast milk. Consider the developmental and health benefits of breastfeeding along with the mother’s clinical need for the drug, and any potential adverse effects on the breastfed infant from the drug or the underlying maternal condition.
