09 Nov, 2026 Five-Year Vutrisiran Data Presented at AHA’s Annual Scientific Sessions 2026 Demonstrate Sustained Benefits of RNAi-Powered TTR Silencing

Long-term results from the HELIOS-B Phase 3 study, including two-year open-label extension (OLE) data, showed that patients treated continuously with vutrisiran had lower rates of the composite of all-cause mortality and recurrent cardiovascular events, better-preserved quality of life, and greater stability of cardiac biomarkers through five years than patients who initiated treatment with vutrisiran approximately three years later, with a safety profile consistent with the double-blind period.  

Cuddy, et al. “2-Year Results From the Open-Label Extension Period of the Phase 3 HELIOS-B Study of Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy” 

A post hoc analysis of 48 patients enrolled in HELIOS-B who underwent cardiopulmonary exercise testing (CPET) with concurrent stress echocardiography indicated that patients treated with vutrisiran had preserved exercise capacity and chronotropic response, improved ventilatory efficiency, and showed favorable changes in measures of right ventricular function over two years compared with placebo.  

Thillainathan, et al. “Effects of Vutrisiran on Cardiopulmonary Response to Exercise in ATTR-CM: The HELIOS-B CPET and Stress Echocardiography Substudy”  

Real-world evidence from the ConTTRibute study evaluated demographics and disease characteristics by sex in ATTR amyloidosis through echocardiographic measures indexed to body surface area (BSA), suggesting that standard echocardiographic thresholds derived from predominantly male cohorts may contribute to delayed disease recognition in women. 

Shah, et al. “Sex Differences at Diagnosis Among Patients With Hereditary or Wild-Type Transthyretin Amyloidosis: Analysis From the ConTTRibute Study”