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vutrisiran
N07XX18
Forms & Strengths
- Subcutaneous solution: 25 mg/0.5 mL in a single-dose pre-filled syringe
Adult Dosing
- 25 mg administered subcutaneously once every 3 months
Pediatric Dosing
- Safety and efficacy in pediatric patients have not been established
Indications
- Treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults
Mechanism of Action
- Double-stranded small interfering ribonucleic acid (siRNA) that targets mutant and wild-type transthyretin (TTR) messenger RNA (mRNA)
- Causes degradation of TTR mRNA through RNA interference, reducing the synthesis of TTR protein
Contraindications
- History of severe hypersensitivity reaction to vutrisiran
Adverse Reactions
- Arthralgia
- Pain in extremity
- Dyspepsia
- Decreased blood vitamin A levels
Drug Interactions
- No formal clinical drug-interaction studies have been performed
- Potential for reduction in serum vitamin A levels; supplementation may be required
Curated Content: Needs Vetting Before Put to Clinical Use