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 effects:
Increased dystrophin production.
Pharmacokinetics⬆⬇
Absorption: IV administration results in complete bioavailability.
Distribution: Widely distributed.
Metabolism/Excretion: Not hepatically metabolized; excreted renally.
Half-Life: 3–4 hr.
Time/Action Profile⬆⬇
(plasma concentrations)
ROUTE
ONSET
PEAK
DURATION
IV
unknown
1 hr
unknown
Contraind./Precautions⬆⬇
Contraindicated in:
None.
Use Cautiously in:
OB: Safety not established in pregnancy;
Lactation: Safety not established in breastfeeding.
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.
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⬆⬇
Explain purpose of eteplirsen to patient.
Instruct patient and parents to notify health care professional immediately if signs and symptoms of hypersensitivity reactions occur.
Rep: Advise females of reproductive potential to notify health care professional if pregnancy is planned or suspected or if breastfeeding.
