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Eteplirsen

Pronunciation

(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

Pharmacology

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.

Distribution

Vdss: 600 mL/kg

Excretion

Renal

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.

Contraindications

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

Dosing: Adult

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

Dosing: Pediatric

Refer to adult dosing.

Dosing: Renal Impairment

There are no dosage adjustments provided in the manufacturer’s labeling (has not been studied).

Dosing: Hepatic Impairment

There are no dosage adjustments provided in the manufacturer’s labeling (has not been studied).

Reconstitution

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.

Administration

Administer by IV infusion over 35 to 60 minutes and complete infusion within 4 hours of dilution. 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.

Storage

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.

>10%:

Central nervous system: Equilibrium disturbance (38%)

Dermatologic: Contact dermatitis (25%), excoriation (≥10%), skin rash (≥10%)

Gastrointestinal: Vomiting (38%)

Hematologic & oncologic: Bruise (≥10%)

Local: Catheter pain (≥10%)

Neuromuscular & skeletal: Arthralgia (≥10%)

Respiratory: Upper respiratory tract infection (≥10%)

<1% (Limited to important or life-threatening): Facial flushing (transient; on infusion day), flushing (on infusion day), increased body temperature (transient; on infusion day)

Warnings/Precautions

There are no warnings listed in the manufacturer’s labeling.

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 signs of common cold. Have patient report immediately to prescriber 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.

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