HELIOS-B: Health benefits of vutrisiran for ATTR-CM may increase over time

Vutrisiran decreased the risk for all-cause and cardiovascular mortality in patients with transthyretin amyloid cardiomyopathy (ATTR-CM). Moreover, new data from the HELIOS-B study suggests that the beneficial effects of vutrisiran may increase over time.

In an updated data cut of the HELIOS-B trial, Prof. Marianna Fontana (University College London, UK) specifically looked at all-cause and cardiovascular mortality at 42 months of follow-up [1]. The trial had randomised patients with ATTR-CM 1:1 to vutrisiran, a subcutaneously administered RNA interference therapeutic agent blocking the production of hepatic transthyretin, or a placebo. After 36 months, participants could enter the open-label extension study of HELIOS-B. “At the time of the primary analysis, we had 42-month data from 42.4% of the ongoing participants [2],” explained Prof. Fontana. “Now, we have 42-month data from 96.3% of the ongoing participants (n=653).”

Vutrisiran established a risk reduction of 36% in all-cause mortality compared with placebo (HR 0.64; 95% CI 0.46–0.88; P=0.007). The effect was comparable in the subgroup of participants who did not receive any other therapy besides the study drug (n=396; HR 0.61; 95% CI 0.42–0.90). Likewise, vutrisiran reduced the risk of cardiovascular mortality by 33% in the overall population and by 36% in the monotherapy population. Other cardiovascular events, such as cardiovascular hospitalisation, hospitalisation for heart failure, and urgent heart failure visits, were also less common in participants on vutrisiran than those on placebo.

“These data reinforce the positive results from the HELIOS-B primary analysis and further demonstrate the beneficial effect of vutrisiran on mortality risk and cardiovascular health for patients with ATTR-CM,” concluded Prof. Fontana.

1. Witteles R, et al. Vutrisiran reduces all-cause mortality, cardiovascular mortality and cardiovascular events in patients with transthyretin amyloid cardiomyopathy: analysis from the HELIOS-B trial. Hottest trials and trial updates (1), Heart Failure 2025, 17–20 May, Belgrade, Serbia.
2. Fontana M, et al. N Engl J Med 2025;392:33-44.

Medical writing support was provided by Robert van den Heuvel.
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Posted on 20 May 202517 July 2025