Comparative efficacy and safety of acoramidis versus tafamidis in patients with transthyretin amyloid cardiomyopathy: an anchored matching-adjusted indirect comparison

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ID: 331974
2026
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Abstract
Abstract Aims Phase 3 trials have shown that acoramidis and tafamidis reduce the risk of all-cause mortality (ACM) and cardiovascular-related hospitalization (CVH) versus placebo in patients with transthyretin amyloid cardiomyopathy (ATTR-CM). Heterogeneity across trials and imbalances in treatment effect modifiers make unadjusted indirect comparisons of treatment outcomes inappropriate. We assessed the efficacy of acoramidis and tafamidis using population-adjusted indirect comparisons. Methods and Results We used matching-adjusted indirect comparison (MAIC) to assess the comparative efficacy of acoramidis and tafamidis 80 mg on reducing ACM and CVH over 30 months. We utilized individual patient data from ATTRibute-CM (acoramidis; NCT03860935) and published aggregate data from ATTR-ACT (tafamidis; NCT01994889) and adjusted for population differences. After adjusting for treatment effect modifiers and excluding data after initiation of concomitant tafamidis use in ATTRibute-CM, the effective sample size for ATTRibute-CM was 298 (acoramidis, n = 209; placebo, n = 89). In this MAIC model, acoramidis was associated with a 34% relative risk reduction in the cumulative frequency of CVH versus tafamidis (relative risk ratio 0.66; 95% CI 0.46–0.95). No statistically significant difference in ACM was observed between acoramidis and tafamidis, although the point estimate numerically favoured acoramidis (HR 0.72; 95% CI 0.41–1.26). No statistically significant differences were identified in the indirect comparisons of adverse events between acoramidis and tafamidis. Conclusion In an anchored MAIC, acoramidis was associated with improved outcomes, reflected by a lower risk of CVH versus tafamidis. In the absence of head-to-head data, these findings may offer important insights regarding the relative effectiveness of newer treatments in ATTR-CM.
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openalex_W7221187407 Use this key to autocite in the manuscript while using SciMatic Manuscript Manager or Thesis Manager
Authors
Journal European Heart Journal Open
Year 2026
DOI
10.1093/ehjopen/oeag167
URL
Keywords Keywords not found

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