Delivery 7–10 daysAmvuttra (vutrisiran) 25 mg injection
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Description
💊 Prescription small interfering RNA therapeutic that silences transthyretin production in the liver.
▪︎ Vutrisiran 25 mg/0.5 mL single-dose prefilled syringe, given subcutaneously once every three months
▪︎ Manufacturer: Alnylam Pharmaceuticals
▪︎ FDA approval: June 2022 for polyneuropathy in hereditary ATTR amyloidosis; extended to amyloid cardiomyopathy in March 2025
🔬 How it works: a gene is a blueprint, and the cell builds protein from a working copy — messenger RNA. A small interfering RNA recognises that copy and marks it for destruction, so the protein is never made. A GalNAc carbohydrate tag delivers the molecule almost exclusively to liver cells, which is where transthyretin is produced, and levels fall by roughly 80%. This is the opposite of a stabiliser: rather than holding the protein together, it prevents its production. 📊 Evidence: HELIOS-B (NEJM 2025) in 655 patients — death from any cause plus recurrent cardiovascular events at a hazard ratio of 0.72 (p=0.01), death from any cause through 42 months at 0.65 (p=0.01), 26.5 m better six-minute walk distance and 5.8 points better KCCQ (both p<0.001). About 40% were already on tafamidis and still benefited.
⚠️ Prescription-only, prescribed by a cardiologist or neurologist after the diagnosis is confirmed and light-chain amyloidosis excluded. Vitamin A levels fall as a consequence of the mechanism, so supplementation at a doctor-specified dose is advised and night-vision complaints are taken seriously. The drug cuts off new amyloid but does not remove what is already deposited.
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