Navigating Therapeutic Pathways and Transforming Care in ATTR-CM - Episode 8
Evaluating Efficacy and Safety of Vutrisiran in ATTR-CM from the HELIOS-B Trial
The panel reviews the HELIOS-B Phase 3 trial evaluating vutrisiran, a transthyretin silencer, in patients with ATTR-CM. Participants were randomized to receive vutrisiran or placebo, with approximately 40% already taking tafamidis at trial entry, particularly in the United States. The study was designed with predefined primary and secondary endpoints, assessing outcomes both in the overall population and in those not on baseline tafamidis. The trial demonstrated statistically significant results across all major endpoints, including reductions in all-cause mortality and cardiovascular hospitalizations, with notable benefit observed both in monotherapy and combination-therapy subgroups. The panel highlights the magnitude of treatment effect and notes that these findings supported FDA approval of vutrisiran for ATTR-CM. They also emphasize its favorable safety profile, explaining that common adverse events—such as cardiac failure and arrhythmias—reflect the underlying disease rather than the drug itself. Overall, the experts describe vutrisiran as a meaningful and well-tolerated therapeutic advancement for patients with ATTR-CM.
The panel reviews the HELIOS-B Phase 3 trial evaluating vutrisiran, a transthyretin silencer, in patients with ATTR-CM. Participants were randomized to receive vutrisiran or placebo, with approximately 40% already taking tafamidis at trial entry, particularly in the United States. The study was designed with predefined primary and secondary endpoints, assessing outcomes both in the overall population and in those not on baseline tafamidis. The trial demonstrated statistically significant results across all major endpoints, including reductions in all-cause mortality and cardiovascular hospitalizations, with notable benefit observed both in monotherapy and combination-therapy subgroups.
The panel highlights the magnitude of treatment effect and notes that these findings supported FDA approval of vutrisiran for ATTR-CM. They also emphasize its favorable safety profile, explaining that common adverse events—such as cardiac failure and arrhythmias—reflect the underlying disease rather than the drug itself. Overall, the experts describe vutrisiran as a meaningful and well-tolerated therapeutic advancement for patients with ATTR-CM.
