Alnylam Presents New HELIOS-B Data on Vutrisiran at ACC 2025 Following FDA Approval for ATTR-CM

Alnylam Pharmaceuticals announced new data from its HELIOS-B Phase 3 study evaluating vutrisiran in patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM) at the American College of Cardiology’s 2025 Annual Scientific Session. These results support the recent FDA approval of AMVUTTRA (vutrisiran) as the first RNAi therapeutic approved to reduce cardiovascular death, hospitalizations, and urgent heart failure visits in adults with ATTR-CM.

The study demonstrated vutrisiran’s favorable impact on echocardiographic systolic and diastolic cardiac function, showing improvements in both left and right ventricular performance. Vutrisiran also helped maintain or improve patients’ functional capacity and quality of life, particularly in those with earlier-stage heart failure. The findings highlight the importance of early intervention with vutrisiran and were recently published in the Journal of the American College of Cardiology (JACC).

Vutrisiran, administered quarterly via subcutaneous injection, delivers rapid knockdown of transthyretin to treat ATTR at its source. It is currently approved in over 15 countries for hereditary ATTR polyneuropathy and, more recently in the U.S., for ATTR-CM.