eteplirsen

REMS

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

Contraind./Precautions ⬆ ⬇

Contraindicated in:

None.

Use Cautiously in:

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

Adv. Reactions/Side Effects ⬆ ⬇

Derm: rash

GI: vomiting

MS: arthralgia

Neuro: balance disorder

Resp: upper respiratory tract infection

Misc: HYPERSENSITIVITY REACTIONS

Interactions ⬆ ⬇

Drug-drug:

None reported.

Availability ⬆ ⬇

Solution for injection: 50 mg/mL

Route/Dosage ⬆ ⬇

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

US Brand Names ⬆ ⬇

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.

Classifications ⬆ ⬇

Therapeutic Classification: none assigned

Pharmacologic Classification: antisense oligonucleotides

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

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.

Pronunciation ⬆ ⬇

Code ⬆

NDC Code

60923- Sarepta Therapeutics, Inc.
- 60923-284- Exondys 51
  - 60923-284-10- 1 VIAL, SINGLE-USE in 1 CARTON (60923-284-10) / 10 mL in 1 VIAL, SINGLE-USE

60923- Sarepta Therapeutics, Inc.
- 60923-363- Exondys 51
  - 60923-363-02- 1 VIAL, SINGLE-USE in 1 CARTON (60923-363-02) / 2 mL in 1 VIAL, SINGLE-USE

61434- Catalent Indiana, LLC
- 61434-003-
  - 61434-003-01- 352 VIAL in 1 CASE (61434-003-01) / 2 mL in 1 VIAL

61434- Catalent Indiana, LLC
- 61434-035-
  - 61434-035-01- 288 VIAL in 1 CASE (61434-035-01) / 10 mL in 1 VIAL

63557- Polypeptide Laboratories Inc
- 63557-2072-
  - 63557-2072-1- 400 g in 1 BOTTLE, GLASS (63557-2072-1)

63557- Polypeptide Laboratories Inc
- 63557-2072-
  63557-2072-2- 400 g in 1 BAG (63557-2072-2)

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Indications ⬇