Vutrisiran 25mg/0.5ml Suppliers & Bulk Manufacturers

Available Forms: Injection

Available Strengths: 25mg/0.5ml

Reference Brands: Amvuttra (USA/EU)

Category: Orphan Drugs

Vutrisiran (Amvuttra) is an RNA interference (RNAi) therapeutic used to treat polyneuropathy caused by hereditary transthyretin-mediated amyloidosis (hATTR) in adults. It works by silencing the transthyretin (TTR) gene, reducing the production of abnormal TTR protein that forms amyloid deposits. The medication is administered as a subcutaneous injection (25 mg/0.5 mL) once every 3 months under medical supervision, helping to slow disease progression and improve quality of life in patients with this rare condition.

Product Description:

Vutrisiran is a small interfering RNA that silences the transthyretin gene in the liver. A GalNAc sugar tag directs it specifically to hepatocytes, where the RNA-induced silencing complex degrades transthyretin messenger RNA, cutting production of the protein by around eighty percent. In hereditary and wild-type transthyretin amyloidosis, that protein misfolds and deposits as amyloid in nerves and heart muscle — reducing the supply of it at source is a different strategy from stabilising what is already circulating.

It is used for the polyneuropathy of hereditary ATTR amyloidosis and, following later trial results, for transthyretin amyloid cardiomyopathy, where it reduced death and cardiovascular events. Dosing is a subcutaneous injection once every three months, which is a marked practical advantage over the earlier siRNA requiring three-weekly infusions. Because transthyretin carries retinol-binding protein, vitamin A supplementation is required, and injection site reactions and arthralgia are the common adverse effects.

Sourcing notes. Originator product, on patent, no generic. Quarterly dosing means it is administered in clinic rather than at home in most systems. Which indications are authorised differs by market, so confirm locally. It competes with TTR stabilisers and with other silencing therapies.