eteplirsen

General

Genetic Implications: Genetic Implications

Pronunciation:
e-tep-lir-sen

Trade Name(s)

  • Exondys 51

Ther. Class.
none assigned

Pharm. Class.
temporary class antisense oligonucleotides

Indications

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

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.

Therapeutic Effect(s):

Increased dystrophin production.

Pharmacokinetics

Absorption: IV administration results in complete bioavailability.

Distribution: Widely distributed.

Metabolism and Excretion: Not hepatically metabolized; excreted renally.

Half-life: 3–4 hr.

TIME/ACTION PROFILE (plasma concentrations)

ROUTEONSETPEAKDURATION
IVunknown1 hrunknown

Contraindication/Precautions

Contraindicated in:

  • None.

Use Cautiously in:

OB: Lactation: Safety and effectiveness not established

Adverse Reactions/Side Effects

Derm: rash

GI: vomiting

Neuro: balance disorder

MS: arthralgia

Resp: upper respiratory tract infection

Misc: HYPERSENSITIVITY REACTIONS (INCLUDING RASH, URTICARIA, PYREXIA, FLUSHING, DYSPNEA, BRONCHOSPASM, AND HYPOTENSION)

* CAPITALS indicate life-threatening.
Underline indicate most frequent.

Interactions

Drug-Drug

None noted.

Route/Dosage

IV Children 30 mg/kg once weekly.

Availability

Solution for IV infusion (must be diluted): 50 mg/mL

Assessment

  • Monitor for signs and symptoms of hypersensitivity reactions (rash, urticaria, pyrexia, flushing, cough, dyspnea, bronchospasm, hypotension) during infusion. Treat symptomatically. Consider slowing or interrupting infusion.

Potential Diagnoses

Implementation

IV Administration

  • Apply topical anesthetic cream to infusion site prior to infusion. Flush IV line with 0.9% NaCl before and after infusion.
  • Intermittent Infusion: Allow vials to reach room temperature before using; mix by inverting 2–3 times, do not shake. Solution is clear and colorless with some opalescence; do not administer solutions that are discolored or contain particulate matter. Diluent: Withdraw dose from vials and dilute with 0.9% NaCl for a total volume of 100–150 mL. Complete infusion within 4 hrs. Diluted solution stable for up to 24 hrs if refrigerated. Do not freeze.
  • Rate:Infuse over 35 to 60 min.
  • Y-Site Incompatibility: Do not infuse other medication concomitantly in same IV access line with eteplirsen.

Patient/Family Teaching

Instruct patient and parents to notify health care professional immediately if signs and symptoms of hypersensitivity reactions occur.

Evaluation/Desired Outcomes

Increase in dystrophin levels in muscle tissues.

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Anesthesia Central is an all-in-one web and mobile solution for treating patients before, during, and after surgery. This collection of drug, procedures and test information is derived from Davis’s Drug, MGH Clinical Anesthesia Procedures, Pocket Guide to Diagnostic Tests, and MEDLINE Journals. .