Vutrisiran
Contents
Description
Vutrisiran is a subcutaneously administered RNAi therapeutic targeting transthyretin (TTR) for hereditary transthyretin-mediated amyloidosis (hATTR) with polyneuropathy. It uses enhanced stabilization chemistry (ESC) allowing quarterly dosing, a significant improvement over monthly administration. The drug demonstrated robust TTR reduction and clinical benefit in the HELIOS-A phase 3 trial.
Indications & Therapeutic Use
Hereditary transthyretin-mediated amyloidosis (hATTR) with polyneuropathy in adults
Global Availability (6 countries)
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Available Through
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| Generic Name | vutrisiran |
| Brands | 1 brand available |
| Active Ingredient | vutrisiran sodium |
| Drug Class | Hereditary transthyretin-mediated amyloidosis (hATTR) with polyneuropathy in adults |
| Manufacturer | Alnylam Pharmaceuticals |
| Dosage Forms | Subcutaneous injection, 25mg/0.5mL pre-filled syringe |
| Medical Code | N07XX14 |
| Orphan Status | Yes — Orphan Drug |
| Cold Chain | Required |
| Lead Time | 18 days |
| Reg. Status | FDA Approved (2022), EMA Approved (2022) |
| Clinical Trial | NCT03759379 |
| Countries | 6 countries |