Summary

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• This summary is based on the review of one randomized controlled trial. ^[1]
• Primary Effectiveness Findings: Acoramidis significantly outperformed placebo in the composite primary outcome with a win ratio of 1.8 (95% confidence interval (CI), 1.4 to 2.2; P < 0.001), favoring acoramidis in 63.7% of pairwise comparisons versus 35.9% for placebo. Death from any cause and cardiovascular-related hospitalizations accounted for 58% of the outcomes.
• Secondary Effectiveness Components: N-terminal prohormone of brain natriuretic peptide (NT-proBNP) levels exhibited the highest proportion of favorable pairwise comparisons for acoramidis (23.3% wins vs. 7.0% losses). Outcomes for 6-minute walk distance, Kansas City Cardiomyopathy Questionnaire-Overall Summary, and serum transthyretin (TTR) level were mentioned but not detailed in the study.
• The overall incidence of adverse events was comparable between the acoramidis group (98.1%) and the placebo group (97.6%), while serious adverse events occurred in 54.6% of the acoramidis group and 64.9% of the placebo group.
• The study does not specify particular safety concerns or adverse effects unique to specific population types or subgroups.
• There is no population types or subgroups information available in the reviewed studies.