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Medically reviewed by Last updated on May 5, 2020.


(e TEP lir sen)

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)

Brand Names: U.S.

  • Exondys 51

Pharmacologic Category

  • Antisense Oligonucleotide


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.

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 noncoring 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. Do not use if solution in vial is cloudy, discolored, or contains extraneous particulate matter other than trace amounts of small, white to off-white, amorphous particles.


IV: Administer by IV infusion over 35 to 60 minutes through a 0.2-micron inline filter; 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

The following adverse drug reactions and incidences are derived from product labeling unless otherwise specified.

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

What is this drug used for?

• It is used to treat Duchenne muscular dystrophy (DMD).

All drugs may cause side effects. However, many people have no side effects or only have minor side effects. Call your doctor or get medical help if any of these side effects or any other side effects bother you or do not go away:

• Vomiting

• Joint pain

• Bruising

• Injection site pain or irritation

• Common cold symptoms

WARNING/CAUTION: Even though it may be rare, some people may have very bad and sometimes deadly side effects when taking a drug. Tell your doctor or get medical help right away if you have any of the following signs or symptoms that may be related to a very bad side effect:

• Flushing

• Cough

• Shortness of breath

• Severe dizziness

• Passing out

• Change in balance

• Signs of an allergic reaction, like rash; hives; itching; red, swollen, blistered, or peeling skin with or without fever; wheezing; tightness in the chest or throat; trouble breathing, swallowing, or talking; unusual hoarseness; or swelling of the mouth, face, lips, tongue, or throat.

Note: This is not a comprehensive list of all side effects. Talk to your doctor if you have questions.

Consumer Information Use and Disclaimer: This information should not be used to decide whether or not to take this medicine or any other medicine. Only the healthcare provider has the knowledge and training to decide which medicines are right for a specific patient. This information does not endorse any medicine as safe, effective, or approved for treating any patient or health condition. This is only a limited summary of general information about the medicine's uses from the patient education leaflet and is not intended to be comprehensive. This limited summary does NOT include all information available about the possible uses, directions, warnings, precautions, interactions, adverse effects, or risks that may apply to this medicine. This information is not intended to provide medical advice, diagnosis or treatment and does not replace information you receive from the healthcare provider. For a more detailed summary of information about the risks and benefits of using this medicine, please speak with your healthcare provider and review the entire patient education leaflet.

Further information

Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.

Frequently Asked Questions