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

Contraindicated in:

• None.

Use Cautiously in:

• OB: Safety not established in pregnancy
• Lactation: Safety not established in breastfeeding.

Derm: rash.

GI: vomiting.

MS: arthralgia.

Neuro: balance disorder.

Resp: upper respiratory tract infection.
Misc: HYPERSENSITIVITY REACTIONS .

Drug-Drug:

• None reported.

• Solution for injection: 50 mg/mL;

• IV (Children): 30 mg/kg once weekly.

Exondys 51

• 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.

• Increased dystrophin production.

Therapeutic Classification: n/a

Pharmacologic Classification: antisense oligonucleotides

Absorption: IV administration results in complete bioavailability.

Distribution: Widely distributed.

Metabolism/Excretion: Not hepatically metabolized; excreted renally.

Half-life: 3–4 hr.

(plasma concentrations)

• Explain purpose of eteplirsen to patient.
• Instruct patient and parents to notify health care professional immediately if signs and symptoms of hypersensitivity reactions occur.
• Advise females of reproductive potential to notify health care professional if pregnancy is planned or suspected or if breastfeeding.

e-TEP-lir-sen