Vutrisiran in Transthyretin Amyloidosis: A Narrative Review.

Abstract

This report describes vutrisiran (Amvuttra^®), a novel double-stranded small interfering ribonucleic acid (siRNA)-N-acetylgalactosamine (GalNAc) conjugate. A narrative literature review was conducted using a secondary database, PubMed, from 2018 to 2025. Articles were limited to those written in English. Inclusion criteria consisted of randomized controlled trials and systematic reviews that analyzed the use of vutrisiran to treat amyloidosis. Other drug information resources, including the package inserts and UpToDate, were also utilized for general information. Two phase 3 publications were included in this report. Vutrisiran significantly improved neuropathy, quality of life, and functional measures in patients with hereditary transthyretin amyloidosis with polyneuropathy. Additionally, vutrisiran reduced the risk of death and recurrent cardiovascular events, preserved functional capacity and quality of life, and slowed New York Heart Association (NYHA) class progression compared with placebo. A favorable safety profile was also found in landmark trials. Vutrisiran is a therapeutic option for amyloidosis, directly targeting the underlying pathogenic mechanism through RNA interference. Clinical trials have demonstrated a reduction in transthyretin protein levels, slowing neuropathic and cardiac disease progression, and improving functional and quality-of-life outcomes. Ongoing research on long-term outcomes is still to be elucidated.