golodirsen

REMS

Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping.

Contraind./Precautions ⬆ ⬇

Contraindicated in:

Hypersensitivity.

Use Cautiously in:

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

Adv. Reactions/Side Effects ⬆ ⬇

CV: tachycardia

EENT: nasopharyngitis

GI: abdominal pain, nausea, vomiting, constipation, diarrhea

GU: nephrotoxicity

Local: injection site pain

MS: back pain

Neuro: headache, dizziness

Resp: cough

Misc: falls, fever, HYPERSENSITIVITY REACTIONS (INCLUDING ANAPHYLAXIS)

Interactions ⬆ ⬇

Drug-drug:

None reported.

Availability ⬆ ⬇

Solution for injection: 50 mg/mL

Route/Dosage ⬆ ⬇

IV (Adults and 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 to extravascular tissues.

Metabolism/Excretion: Not hepatically metabolized. Primarily excreted unchanged in urine.

Half-Life: 3.4 hr.

Time/Action Profile ⬆ ⬇

(plasma concentrations)

ROUTE	ONSET	PEAK	DURATION
IV	unknown	unknown	unknown

Patient/Family Teaching ⬆ ⬇

Explain purpose and side effects of medication to patient. Advise patient to read Patient Information before starting therapy.
Advise patient to notify health care professional of all Rx or OTC medications, vitamins, or herbal products being taken and to consult health care professional before taking other medications.
Explain need for continued medical follow-up to assess effectiveness and possible side effects of medication. Periodic lab tests will be needed. Emphasize importance of regular lab tests to monitor for renal toxicity.
Advise patient to notify health care professional immediately if signs and symptoms of hypersensitivity reactions occur (rash, hives, fever, itchiness, skin peeling).
Rep: Advise patient to notify health care professional if pregnancy is planned or suspected or if breastfeeding.

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Indications ⬇