Consistent efficacy of vutrisiran in women and men with ATTR-CM

ESC Heart Failure 2026 – In a prespecified analysis of HELIOS-B, vutrisiran reduced the risk of all-cause mortality or recurrent CV events in women and men with ATTR-CM.

This summary is based on the presentation of Josephine Mansell, MD (London, UK) at the ESC Heart Failure Congress 2026 - Consistent efficacy of vutrisiran across sexes in ATTR-CM. HELIOS-B trial – Prespecified sex analysis.

Introduction and methods

There is evidence that women with ATTR-CM have a distinct baseline clinical phenotype compared with men. However, women are underrepresented in clinical trials and clinical registries. This limits our ability to investigate, understand and characterize the potential differences between sexes. There is a need for more sex-specific analysis of trial outcomes in ATTR-CM.

HELIOS-B (A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy) was a global, multicenter, placebo-controlled, double-blind, phase 3 RCT in which 654 patients with a confirmed diagnosis of ATTR-CM (wild-type or hereditary) and history of symptomatic HF were randomized to subcutaneous vutrisiran 25 mg or placebo every 3 months for up to 36 months.

In HELIOS-B, 49 patients (7.5%) were women. Variant ATTR-CM was more common in women compared with men (42.9% vs. 9.1%; P<0.001).

The primary endpoint was the composite outcome of all-cause mortality or recurrent CV events during the double-blind period (month 33-36) in both the overall population and the monotherapy population (patients not on tafamidis at baseline).

Main results

• Compared with men, women had reduced functional capacity, worse heath status as measured with KCCQ, lower levels of troponin I, and less atrial arrhythmia.
• Compared with placebo, vutrisiran reduced the risk of all-cause mortality or recurrent CV events in women (HR: 0.59; 95%CI: 0.26-1.30) and men (HR:0.71; 95%CI: 0.54-0.94; P for interaction=0.66).

Conclusion

In this prespecified analysis of HELIOS-B, vutrisiran reduced the risk of risk of all-cause mortality or recurrent CV events in women and men. Dr. Mansell acknowledges that the small number of female participants in this study limits the statistical precision of this analysis and therefore the results should be interpreted with caution.

- Our reporting is based on the information provided at the ESC Heart Failure Congress 2026 -