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Eteplirsen (Monograph)

Brand name: Exondys 51
Drug class: Antisense Oligonucleotides
Chemical name: 5′-[P-[4-[[2-[2-(2-hydroxyethoxy)ethoxy]ethoxy]carbonyl]-1-piperazinyl]-N,N-dimethylphosphonamidate] [P-deoxy-P-(dimethylamino)](2′,3′-dideoxy-2′,3′-imino-2′,3′-seco)(2′a→ 5′)(C-m5U- C-C-A-A-C-A-m5U-C-A-A-G-G-A-A-G-A-m5U-G-G-C-A-m5U-m5U-m5U-C-m5U-A-G) RNA
Molecular formula: C364H569N177O122P30
CAS number: 1173755-55-9

Medically reviewed by on Apr 16, 2024. Written by ASHP.


Antisense oligonucleotide that binds to exon 51 of dystrophin premessenger RNA (pre-mRNA).

Uses for Eteplirsen

Duchenne Muscular Dystrophy

Management of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation of the dystrophin gene that is amenable to skipping of exon 51 (designated an orphan drug by FDA for use in this condition).

Accelerated approval based on increased dystrophin production (a surrogate marker of response). Continued approval may be contingent on verification of clinical benefit (e.g., improvement in motor function) in confirmatory studies.

Approximately 13–14% of patients with DMD have specific mutations of the dystrophin gene amenable to exon 51 skipping.

Eteplirsen Dosage and Administration


Administer by IV infusion. Use an inline 0.2-µm filter.

IV Administration

For solution compatibility information, see Compatibility under Stability.

Consider pretreatment of the infusion site with topical anesthetic cream.

Do not mix or administer simultaneously with other drugs. Flush IV line with 0.9% sodium chloride injection before and after infusion.

If a dose is missed, administer missed dose as soon as possible.

Vials are for single use only; discard unused portions since the concentrate contains no preservatives.


Must dilute injection concentrate prior to IV infusion.

Visually inspect vials. The injection concentrate should be a clear to slightly opalescent and colorless solution that may contain trace amounts small, white to off-white amorphous particles.

Determine number of vials needed based on patient’s body weight and recommended dose. Allow vials to warm to room temperature. Mix contents by gently inverting vials 2–3 times; do not shake.

Withdraw appropriate volume of injection concentrate from appropriate number of vials using a syringe with a 21-gauge or smaller noncoring needle and dilute with 0.9% sodium chloride injection to a total volume of 100–150 mL.

Visually inspect diluted solution; do not use if it is cloudy, discolored, or contains extraneous particulate matter other than trace amounts small, white to off-white amorphous particles.

Administer immediately after dilution, completing administration within 4 hours. If immediate administration not possible, may store diluted solution at 2–8°C for up to 24 hours. Do not freeze.

Rate of Administration

Infuse IV over 35–60 minutes. If hypersensitivity reaction occurs, consider slowing infusion or interrupting therapy. (See Sensitivity Reactions under Cautions.)


Pediatric Patients

Duchenne Muscular Dystrophy

30 mg/kg once weekly.


Duchenne Muscular Dystrophy

30 mg/kg once weekly.

Special Populations

No special population dosage recommendations at this time.

Cautions for Eteplirsen



Sensitivity Reactions

Hypersensitivity reactions (e.g., rash, urticaria, pyrexia, flushing, cough, dyspnea, bronchospasm, hypotension) reported.

If hypersensitivity reaction occurs, initiate appropriate medical treatment and consider slowing the eteplirsen IV infusion or interrupting therapy.

Specific Populations


Not studied in female patients. Not known whether eteplirsen is associated with risk if used during pregnancy.


Not studied in female patients. Not known whether eteplirsen is distributed into milk, affects milk production, or affects the breast-fed infant.

Consider known benefits of breast-feeding along with mother's clinical need for eteplirsen and any potential adverse effects of the drug or disease on the infant.

Pediatric Use

Eteplirsen is labeled for use in certain pediatric patients with DMD. (See Uses.)

In juvenile animal studies, renal tubular necrosis with associated clinical pathology changes and changes in bone density observed. No effects on male reproductive system, neurobehavioral development, or immune function.

Geriatric Use

No experience in geriatric patients; DMD is generally a disease of children and young adults.

Hepatic Impairment

Not studied in patients with hepatic impairment.

Renal Impairment

Not studied in patients with renal impairment.

Common Adverse Effects

Balance disorder, vomiting, contact dermatitis.

Drug Interactions

In vitro data indicate low potential for drug interactions.

Drugs Affecting or Metabolized by Hepatic Microsomal Enzymes

Does not appear to be metabolized by hepatic microsomal enzymes.

Does not substantially inhibit CYP isoenzymes 1A2, 2B6, 2C8, 2C9, 2C19, 2D6, or 3A4/5 in vitro.

Does not induce CYP2B6 or 3A4. Induces CYP1A2, but to a substantially lesser degree than the prototype CYP1A2 inducer (i.e., omeprazole).

Drugs Affecting or Affected by Membrane Transporters

Not a substrate or inhibitor of organic anion transporter (OAT) 1 or 3, organic cation transporter (OCT) 1 or 2, organic anion transporting protein (OATP) 1B1 or 1B3, P-glycoprotein (P-gp), breast cancer resistance protein (BCRP), multidrug resistance protein (MRP) 2, or bile salt export pump (BSEP).

Eteplirsen Pharmacokinetics



Following single or multiple IV infusions at doses of 0.5 to 50 mg/kg per week, peak plasma concentrations observed near the end of infusion at 1.1–1.2 hours.

Exhibits dose proportional, linear pharmacokinetics; no substantial drug accumulation following weekly infusion.



Not known whether distributed into human milk.

Plasma Protein Binding




Does not appear to be metabolized by hepatic microsomal enzymes.

Elimination Route

Renal clearance accounts for approximately two-thirds of the administered dose within 24 hours of IV infusion.


3–4 hours.




Injection concentrate

2–8°C; do not freeze. Protect from light; store in original carton until use.

Diluted solution: May store at 2–8°C for up to 24 hours; do not freeze.



Solution Compatibility


Sodium chloride 0.9%


Advice to Patients


Excipients in commercially available drug preparations may have clinically important effects in some individuals; consult specific product labeling for details.

Please refer to the ASHP Drug Shortages Resource Center for information on shortages of one or more of these preparations.



Dosage Forms


Brand Names



For injection concentrate, for IV infusion

50 mg/mL

Exondys 51


AHFS DI Essentials™. © Copyright 2024, Selected Revisions April 26, 2021. American Society of Health-System Pharmacists, Inc., 4500 East-West Highway, Suite 900, Bethesda, Maryland 20814.

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