Section 12 · Monograph
Patisiran
Reviewed Nov 2026FDA label
OnpattrosiRNA (TTR-directed) · IV
- Forms
- IV: 10mg/5mL (2mg/mL) vial
- Dose
- Under 100kg: 0.3mg/kg IV every 3 weeks | 100kg or more: 30mg every 3 weeks; premedicate
- MOA
- Silences hepatic transthyretin (TTR) synthesis
Indications
- Polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adultsFDA-labeled
Mechanism of Action
- Double-stranded siRNA in a lipid complex that targets the 3' untranslated region of mutant and wild-type TTR mRNA in the liver, reducing TTR production
Full Dosing
Adult / usual
- Under 100kg: 0.3mg/kg IV every 3 weeks; 100kg or more: 30mg IV every 3 weeks; infuse over about 80 minutes through an ambulatory pump and filter
- Premedicate at least 60 minutes before each infusion with an IV corticosteroid (e.g., dexamethasone 10mg), oral acetaminophen 500mg, and IV H1 and H2 blockers
- Vitamin A supplementation at the recommended daily allowance
- Missed dose: if given within 3 days, continue the original schedule; otherwise continue every 3 weeks thereafter
Contraindications
- None listed in labeling
Drug Interactions
- No clinically significant interactions expected
Adverse Effects
- Upper respiratory tract infections
- Infusion-related reactions (flushing, back pain, nausea)
- Reduced serum vitamin A levels
Monitoring Parameters
- Infusion reactions
- Ocular symptoms of vitamin A deficiency
- Neuropathy scales
Pregnancy & Lactation
- Reduced vitamin A may harm the fetus; animal developmental toxicity at maternally toxic doses
- No information on presence in human milk