Transthyretin amyloid cardiomyopathy (ATTR-CM) is a rare and life-threatening disease characterized by the accumulation of abnormal transthyretin protein in the heart tissue. BridgeBio Pharma has recently published their successful Phase 3 trial results of acoramidis, a potential treatment for ATTR-CM, in the New England Journal of Medicine. This article provides a comprehensive overview of the study and its outcome.
Study Design and Findings
The phase 3 study, named ATTRibute-CM, aimed to evaluate the efficacy of acoramidis in improving patient outcomes with ATTR-CM. The trial enrolled a significant number of patients and utilized a hierarchical analysis that considered two primary endpoints as measures of treatment effect: all-cause mortality (ACM) and the frequency of cardiovascular-related hospitalization (CVH).
Results from the study demonstrated a highly significant treatment effect of acoramidis on the primary endpoint, with a Win Ratio of 1.8 (p
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