vutrisiran
Amvuttra (vutrisiran) is a small interfering RNA medicine from Alnylam that silences production of transthyretin. It treats the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults, and in the United States also the cardiomyopathy of wild-type or hereditary transthyretin amyloidosis.
This page is based on the official US label for AMVUTTRA and the EMA medicine record for Amvuttra; the mechanism is explained in plain language, and analogies are used only where they stay accurate.
How it works
Explains what the medicine does in the body, not dosing, side effects, or safety.
An analogy
In ATTR amyloidosis the liver keeps printing copies of a faulty protein that clumps into amyloid deposits in nerves and heart. Vutrisiran slips into the liver and quietly shreds the printing instructions, so far less faulty protein is ever made.
In simple terms
Vutrisiran is given by injection every three months. It is a small interfering RNA that switches off the liver's production of transthyretin, the protein that misfolds and builds up as amyloid in nerves and the heart. With less transthyretin made, amyloid deposits form more slowly.
In depth
Vutrisiran is a double-stranded small interfering RNA (siRNA) conjugated to a trivalent N-acetylgalactosamine (GalNAc) ligand for hepatocyte targeting. Inside liver cells it engages the RNA-induced silencing complex and degrades transthyretin (TTR) messenger RNA, sharply reducing circulating TTR, both mutant and wild-type. Lower TTR availability slows amyloid fibril formation in peripheral nerves and myocardium, the basis of its benefit in hATTR polyneuropathy and ATTR cardiomyopathy.
See it in motion
A schematic animation of the mechanism — simplified for clarity, not drawn to scale.
How siRNA silencers switch off a gene
- The liver is making too much of a protein that drives the disease.
- The siRNA enters liver cells and directs the cell's RNA-silencing machinery to the TTR messenger RNA.
- When it finds the target mRNA, the complex cuts it so the protein can no longer be built.
- With less of the harmful protein, the disease process slows.
Target: Transthyretin (TTR) messenger RNA
The liver-produced messenger RNA that carries instructions for making transthyretin, the protein that misfolds and forms amyloid deposits in ATTR amyloidosis.
Approved uses
Approved uses differ by country and change over time. Always follow your local prescribing information.
Approved uses in United States
FDA- Treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults
- Treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality, cardiovascular hospitalizations and urgent heart failure visits
Approved uses in European Union
EMA- Treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult patients with stage 1 or stage 2 polyneuropathy
Official sources
The statements on this page trace back to these records.
- Amvuttra (vutrisiran) — Summary of product characteristics and package leaflet, English version
European Medicines Agency
- openFDA drug label record for vutrisiran (Amvuttra)
U.S. Food and Drug Administration, openFDA
- DailyMed: AMVUTTRA (vutrisiran) official US label
U.S. National Library of Medicine, DailyMed
This page explains how the medicine works — it isn’t the patient leaflet. For anything that applies to you, read the leaflet that came with your medicine or ask your pharmacist.
MedUnfold provides educational content and is not medical advice. Always follow your local prescribing information and healthcare professional.