Early and continuous treatment with transthyretin (TTR) stabilizer acoramidis led to significant improvements in health status and slower functional decline in individuals with transthyretin amyloid cardiomyopathy (ATTR-CM), according to data presented at the 2026 Annual Meeting of the American Association of Heart Failure Nurses (AAHFN 2026) in San Diego, California.
Acoramidis, a highly selective oral TTR stabilizer that achieves near-complete (≥90%) disease stabilization, is approved in the United States, Europe, Japan, Switzerland, and the United Kingdom for the treatment of variant or wild-type ATTR-CM in adults. In the phase 3 ATTRibute-CM study, which enrolled more than 600 patients who were randomized to receive acoramidis hydrochloride at a dose of 800 mg or matching placebo twice daily for 30 months, treatment with acoramidis achieved ATTR-CM disease stabilization, reflected in sustained increases in serum TTR concentrations and stabilized N-terminal pro–B-type natriuretic peptide levels. An interim analysis showed that acoramidis significantly increased serum TTR concentrations early, by day 28 after treatment initiation, and that the increase in concentrations was sustained through month 30 [Maurer MS, et al. J Am Coll Cardiol 2025; 85:1911-23]. The acoramidis-mediated early increase in serum TTR concentrations was independently associated with improved survival. The researchers also assessed Kansas City Cardiomyopathy Questionnaire (KCCQ) scores, which measure a patient's health status, symptom burden, physical limitations, and quality of life caused by heart failure or cardiomyopathy, and found that acoramidis treatment also reduced the decline in heart failure-related health status (as assessed by KCCQ scores) compared with placebo at month 30.