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Generic name: eteplirsen systemic

Brand names: Exondys 51

Dosage Forms

Excipient information presented when available (limited, particularly for generics); consult specific product labeling.

Solution, Intravenous [preservative free]:

Exondys 51: 50 mg/mL (2 mL, 10 mL)


Mechanism of Action

Binds to exon 51 of dystrophin pre-messenger RNA (mRNA), resulting in exclusion of this exon during mRNA processing. Exon skipping allows for production of an internally truncated dystrophin protein.



Vdss: 600 mL/kg



Time to Peak

1.1 to 1.2 hours

Half-Life Elimination

3 to 4 hours

Protein Binding

6% to 17%

Use: Labeled Indications

Duchenne muscular dystrophy: Treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping.


There are no contraindications listed in the manufacturer's labeling.

Dosage and Administration

Dosing: Adult

Duchenne muscular dystrophy: IV: 30 mg/kg once weekly

Dosing: Pediatric

Duchenne muscular dystrophy (DMD): Male children ≥7 years and Adolescents: IV: 30 mg/kg/dose once weekly; in the clinical trials, patients ranged in age from 7 to 13 years (Mendell 2013, Mendell 2016)

Dosing: Adjustment for Toxicity

Hypersensitivity reactions: Consider slowing the infusion or interrupting therapy.


Allow vials to warm to room temperature prior to dilution. Mix contents of each vial by gently inverting 2 or 3 times; do not shake. Use a 21-gauge or smaller non-coring needle to withdraw the calculated volume of eteplirsen and dilute in NS to a total volume of 100 to 150 mL. Administer diluted solution immediately.


IV: Administer by IV infusion over 35 to 60 minutes and complete infusion within 4 hours of dilution. Consider slowing the infusion or interrupting therapy for hypersensitivity reactions. Flush IV access line with NS prior to and after infusion. Do not mix with other medications or infuse other medications concomitantly via the same IV access line. If a dose is missed, administer as soon as possible after the scheduled time.


Store at 2°C to 8°C (36°F to 46°F). Do not freeze. Protect from light and store in the original carton until ready for use. The diluted solution may be stored at 2°C to 8°C (36°F to 46°F) for up to 24 hours; do not freeze. Discard unused portion.

Drug Interactions

There are no known significant interactions.

Adverse Reactions

Incidences include reactions experienced with approved and unapproved doses.


Central nervous system: Equilibrium disturbance (38%)

Dermatologic: Contact dermatitis (25%)

Gastrointestinal: Vomiting (38%)

<1%, postmarketing, and/or case reports: Bronchospasm, cough, dyspnea, fever, flushing, hypersensitivity reaction, hypotension, urticaria


Concerns related to adverse effects:

  • Hypersensitivity: Hypersensitivity reactions (including rash, urticaria, pyrexia, flushing, cough, dyspnea, bronchospasm, and hypotension) have been reported with use; manage appropriately and consider slowing the infusion rate or interrupting therapy.


Pregnancy Considerations

Animal reproduction studies have not been conducted. Eteplirsen has not been studied in females.

Patient Education

  • Discuss specific use of drug and side effects with patient as it relates to treatment. (HCAHPS: During this hospital stay, were you given any medicine that you had not taken before? Before giving you any new medicine, how often did hospital staff tell you what the medicine was for? How often did hospital staff describe possible side effects in a way you could understand?)
  • Patient may experience vomiting, joint pain, bruising, injection site pain or irritation, or common cold symptoms. Have patient report immediately to prescriber flushing, cough, shortness of breath, severe dizziness, passing out, or change in balance (HCAHPS).
  • Educate patient about signs of a significant reaction (eg, wheezing; chest tightness; fever; itching; bad cough; blue skin color; seizures; or swelling of face, lips, tongue, or throat). Note: This is not a comprehensive list of all side effects. Patient should consult prescriber for additional questions.

Intended Use and Disclaimer: Should not be printed and given to patients. This information is intended to serve as a concise initial reference for health care professionals to use when discussing medications with a patient. You must ultimately rely on your own discretion, experience, and judgment in diagnosing, treating, and advising patients.

Source: Wolters Kluwer Health. Last updated March 2, 2019.