patisiran

REMS

Treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis.

Contraind./Precautions ⬆ ⬇

Contraindicated in:

None.

Use Cautiously in:

Moderate or severe hepatic impairment;
Severe renal impairment or end-stage renal disease;
OB: Safety not established in pregnancy;
Lactation: Use while breastfeeding only if potential maternal benefit justifies potential risk to infant;
Pedi: Safety and effectiveness not established in children.

Adv. Reactions/Side Effects ⬆ ⬇

CV: heart block

Derm: erythema

EENT: blurred vision, dry eyes, vertigo, vitreous floaters

GI: dyspepsia

Metab: ↓serum vitamin A concentrations

MS: arthralgia, muscle spasms

Resp: upper respiratory tract infections, dyspnea

Misc: infusion reactions

Interactions ⬆ ⬇

Drug-drug:

None reported.

Availability ⬆ ⬇

Solution for injection: 2 mg/mL

Route/Dosage ⬆ ⬇

IV (Adults ≥100 kg): 30 mg every 3 wk.
IV (Adults <100 kg): 0.3 mg/kg every 3 wk.

US Brand Names ⬆ ⬇

Action ⬆ ⬇

Contains a double-stranded small interfering ribonucleic acid (siRNA) that causes degradation of mutant and wild-type transthyretin (TTR) mRNA. through RNA interference, which results in a reduction of serum TTR protein and TTR protein deposits in tissues.

Therapeutic effects:

Improvement in neuropathy.

Classifications ⬆ ⬇

Therapeutic Classification: none assigned

Pharmacologic Classification: antitransthyretin small interfering rnas

Pharmacokinetics ⬆ ⬇

Absorption: IV administration results in complete bioavailability.

Distribution: Well distributed to tissues.

Metabolism/Excretion: Primarily metabolized by nucleases to nucleotides; <1% excreted in urine as unchanged drug.

Half-Life: 3.2 days.

Time/Action Profile ⬆ ⬇

(serum TTR concentrations)

ROUTE	ONSET	PEAK	DURATION
IV	10–14 days	unknown	unknown

Patient/Family Teaching ⬆ ⬇

Explain purpose of patisiran to patient.
Advise patient to take supplement of vitamin A at recommended daily allowance and to notify health care professional if symptoms of vitamin A deficiency (night blindness) occur.
Advise patient to notify health care professional immediately if signs and symptoms of infusion-related reactions occur.
Rep: Advise patient to notify health care professional if pregnancy is planned or suspected or if breastfeeding. Encourage pregnant patients to enroll in the pregnancy exposure registry that monitors pregnancy outcomes in women exposed to patisiran during pregnancy by calling 1-877-256-9526 or by contacting [email protected].

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Indications ⬇