Alnylam reports positive results for heart drug in pivotal study, likely clearing path to approval
How did vutrisiran perform in patients not taking standard care treatments?
In patients not taking standard care treatments, known as the monotherapy population, vutrisiran reduced the risk by 33% compared to placebo on the composite primary endpoint and cut the risk of all-cause death by 35%.
What is the likely next step for vutrisiran following this study?
Following the positive results from the HELIOS-B Phase 3 study, the likely next step for vutrisiran is to proceed with global regulatory submissions3. Alnylam Pharmaceuticals plans to file a U.S. Supplemental New Drug Application using a Priority Review Voucher, with the aim of obtaining regulatory approval for the treatment of ATTR amyloidosis with cardiomyopathy.
What was the percentage risk reduction of all-cause death by vutrisiran?
Vutrisiran reduced the risk of all-cause death by 36% in the overall population and by 35% in the monotherapy group2. This outcome included up to six months of data from an open-label extension that followed the blinded period of the trial.