RNA interference with vutrisiran reduces the risk of death and recurrent cardiovascular events in patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM). This finding from the HELIOS-B trial was presented at the ESC Congress 2024.

When transthyretin misfolds (owing to variants in TTR or ageing), the protein accumulates as amyloid fibrils in multiple organs, typically resulting in polyneuropathy or an infiltrative cardiomyopathy. The only approved treatment for ATTR-CM is tafamidis, which stabilizes the transthyretin tetramer; however, mortality remains high, and quality of life and functional capacity continue to decline.