In a recent study published in the Journal of the American College of Cardiology, vutrisiran significantly reduced mortality and cardiovascular complications in people with transthyretin amyloidosis with cardiomyopathy (ATTR-CM).
The data comes from a follow-up of the HELIOS-B trial, which included 655 participants with ATTR-CM who were randomly assigned to receive either vutrisiran or a placebo for up to 36 months, followed by an open-label extension. The new study includes data from the 96% of patients who were followed for 42 months.
Vutrisiran is an RNA interference therapeutic which works by preventing the liver from producing too much of the TTR protein; it is administered through a small injection under the skin once every three months.
The results showed that treatment led to a 36% reduction in all-cause mortality and a 33% reduction in death from cardiovascular events. The therapy also lowered the rate of cardiovascular hospitalizations by 25%, heart failure hospitalizations by 33%, and urgent heart failure visits by 46%.
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The benefits of vutrisiran were even more pronounced in individuals who were not taking tafamidis, a medication which delays the progression of ATTR-CM, when the study began. In this group, vutrisiran reduced all-cause mortality by 39% and cardiovascular mortality by 36%. Hospitalizations due to heart complications and heart failure were also reduced by 34% and 38% respectively.
The findings are consistent with the results of the HELIOS-B trial and provide further evidence of vutrisiran’s beneficial effects for people with ATTR-CM, the researchers concluded.
“Overall, our findings provide further evidence that vutrisiran offers an effective treatment option that potentially extends and improves patients’ lives by lowering both the rate of cardiovascular events and the risk of all-cause and cardiovascular mortality,” they wrote. “The ongoing HELIOS-B open-label follow-up of up to an additional 2 years will shed further light on the longer-term benefit of early, deep, and sustained TTR knockdown with vutrisiran.”
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