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Vutrisiran reduced all-cause mortality and recurrent cardiovascular events in a population of patients with transthyretin amyloidosis (ATTR) with cardiomyopathy versus placebo, despite a substantial proportion of the HELIOS-B study population being on tafamidis background therapy.

The HELIOS-B trial investigated the RNA interference therapeutic vutrisiran in 655 participants with ATTR cardiomyopathy. The participants were randomly assigned to vutrisiran or placebo and the primary endpoint was a composite of all-cause mortality and recurrent cardiovascular events at 36 months. “Approximately 40% of the participants were on tafamidis at baseline, raising the bar for vutrisiran to demonstrate its efficacy,” emphasized Prof. Marianna Fontana, MD, PhD, from the University College of London, in the UK¹.

The primary endpoint was met, with a hazard ratio of 0.72, and a P-value of 0.012. “This effect was driven by both components of the primary endpoint,” added Prof. Fontana.  Important secondary endpoints were also in favor of the vutrisiran arm, such as change in the 6-minute walk test at month 30 (LS mean difference 26.46; P=0.00008) and KCCQ-overall score change at month 30 (LS mean difference 5.80; P=0.0008).

The safety profiles of vutrisiran and placebo were comparable, with 67.1% and 61.7% of the participants experiencing serious AEs in the placebo arm and vutrisiran arm, respectively. In addition, there were no AEs that were typical for vutrisiran.

“Vutrisiran has the potential to become a standard-of-care for previously untreated patients with ATTR cardiomyopathy and for those who progress on stabilizing therapies,” concluded Prof. Fontana.

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