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patisiran
N07XX12
Forms & Strengths
- Solution for injection: 0.5 mL filled with 2.5 mg/mL patisiran (1.25 mg patisiran per 0.5 mL)
- Single-dose, prefilled syringes
Adult Dosing
- Recommended dose: 0.3 mg/kg administered via intravenous infusion over approximately 80 minutes
- Administer every 3 weeks
Pediatric Dosing
- Safety and efficacy not established in pediatric patients
Indications
- Treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adults
Mechanism of Action
- Patisiran is a double-stranded RNA molecule that inhibits the production of transthyretin (TTR) through RNA interference (RNAi)
- Inhibition of TTR production reduces the formation of amyloid deposits
Adverse Reactions
- Infusion-related reactions (IRR): 19% of patients
- Neck and upper limb pain
- Fatigue
- Headache
- Vitamin A deficiency
Drug Interactions
- No clinically significant interactions
Curated Content: Needs Vetting Before Put to Clinical Use