Efficacy of Acoramidis in Adults With Transthyretin Amyloid Cardiomyopathy　― Propensity Score Comparison Between a Single-Arm Open-Label Phase 3 Trial in Japan and a Global Double-Blind Phase 3 Trial ―

Key Points

• This analysis aims to compare the efficacy of acoramidis in treating transthyretin amyloid cardiomyopathy (ATTR-CM) between two phase 3 trials.
• Analyzed data from a single-arm open-label phase 3 trial (NCT04622046) in Japan and a global double-blind phase 3 trial (NCT03860935).
• Utilized propensity score matching to compare outcomes between acoramidis-treated patients and placebo patients.
• Found that acoramidis significantly improved efficacy outcomes compared to placebo in matched cohorts over 30 months.