In patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM), the use of vutrisiran resulted in lower risk of cardiovascular events and death from any cause and cardiovascular events compared with placebo, according to findings from the HELIOS-B Trial presented at ESC Congress 2024 in London and simultaneously published in the New England Journal of Medicine. Patients receiving the subcutaneously administered RNA interference therapeutic agent also saw preserved functional capacity and better quality of life, researchers said.

The study randomized 655 patients from 87 in 26 countries to receive either 25 mg of vutrisiran (n=326) or placebo (n=329) once every three months for up to 36 months. All patients had ATTR-CM and more than 75% had heart failure of NYHA class 2 and 40% were taking tafamidis at baseline. The primary endpoint was a composite of death from any cause and recurrent cardiovascular events, while secondary endpoints included death from any cause and change from baseline in results of the 6-minute walk test, and the KCCQ-OS score.

Overall results showed a primary endpoint event occurred in 163 patients in the vutrisiran group compared with 202 in the placebo group. In addition, individuals receiving vutrisiran experienced less of a decline in the distance covered during the 6-minute walk tests, as well as in quality of life determined by the KCCQ-OS score. Researchers noted similar incidences of adverse events between the vutrisiran group and the placebo group (99% vs. 98%, respectively) and no significant differences in serious adverse events (62% vs. 67%, respectively).

"Vutrisiran was highly effective and well tolerated in this contemporary population representative of patients that we see in our clinics, with consistent benefits regardless of background tafamidis therapy," said Primary Investigator Marianna Fontana, MD, PhD. "Our findings indicate that vutrisiran has the potential to become the new standard of care. This trial is also important as it is the first to show the benefit of gene silencers in any type of cardiomyopathy."