Vutrisiran

China [CN]

Vutrisiran Key Information

Generic Name: Vutrisiran  Chinese Name: 武特里西兰 (Wutrisiran)  Trade Name: Amvuttra  CAS Number: 1867157-35-4  Development Code: ALN-TTRsc02  Original Developer: Alnylam Pharmaceuticals

Drug Type and Mechanism of Action

Drug Type: Double-stranded small interfering RNA (siRNA)-GalNAc conjugate, RNA interference (RNAi) therapy

Mechanism of Action:

Precisely targets transthyretin (TTR) mRNA, degrading it through an RNA interference mechanism

Inhibits the synthesis of both mutant and wild-type TTR proteins

Reduces serum TTR levels and decreases TTR amyloid deposition in tissues (nerves, heart)

Utilizes the ESC-GalNAc delivery platform for liver targeting and extended half-life

Core Clinical Applications (2026)

Approved Indications:

Adult hereditary transthyretin amyloidosis polyneuropathy (hATTR-PN)

Adult wild-type or hereditary transthyretin amyloidosis cardiomyopathy (ATTR-CM) (reduces the risk of cardiovascular death, hospitalization, and emergency visits for heart failure)

Dosage Regimen: 25 mg/0.5 mL, subcutaneous injection, once every 3 months

Dosage Form: Pre-filled injection pen/syringe (single dose)

Physicochemical and Formulation Highlights

Appearance: Clear, colorless to pale yellow solution

Solubility: Water-soluble (for injection)

Stability: Store at 2–30℃, do not freeze

Purity: API purity ≥98%, formulation meets sterility and pyrogen-free requirements