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Войти в исследования Circulation reports · 2026
Efficacy and Safety of Acoramidis in Japanese Adults With Symptomatic Transthyretin Amyloid Cardiomyopathy.
СтатьяНазвание и аннотация: Перевод в очереди
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Endo Jin, Tsujita Kenichi, Izumi Chisato, Yano Toshiyuki, Kitaoka Hiroaki, Sekijima Yoshiki, Tahara Nobuhiro, Ishii Shunsuke, Okumura Takahiro, Hashimoto Toru, Fukata Mitsuhiro, Minamino Tohru, Raizner Aileen, Chen Yi, Otsuka Yasushi, Mercuri Michele F, Minamisawa Masatoshi, Ando Yukio
Аннотация
BACKGROUND: This multicenter open-label single-arm 2-part prospective Phase 3 trial (NCT04622046) evaluated the efficacy, safety, pharmacokinetics, and pharmacodynamics of acoramidis, an oral, highly selective transthyretin (TTR) stabilizer, in Japanese patients with wild-type or hereditary symptomatic transthyretin amyloid cardiomyopathy (ATTR-CM).
METHODS AND RESULTS: Twenty-five patients (mean [±SD] age 76.5±6.3 years; 88% men) received acoramidis hydrochloride (800 mg twice daily) for up to 30 months. Primary endpoints were change from baseline to Month 12 in the 6-min walk distance (6MWD) and all-cause mortality and cardiovascular-related hospitalizations over 30 months. No deaths were reported over 30 months. The annualized frequency of cardiovascular-related hospitalizations was 0.133. Clinically meaningful treatment benefits were observed in least-squares mean change from baseline to Month 12 for 6MWD (-3.9 m; 95% confidence interval [CI] -22.9, 15.1 m) and to Month 30 for 6MWD (-36.2 m; 95% CI -58.5 to -13.9 m), Kansas City Cardiomyopathy Questionnaire overall score (-7.0; 95% CI -14.7, 0.8), TTR levels (9.4 mg/dL; 95% CI 6.7, 12.0 mg/dL), and median N-terminal pro B-type natriuretic peptide levels (169.5 pg/mL; interquartile range -557.0, 700.0 pg/mL). The median TTR stabilization (fluorescent probe exclusion assay) was 95.6%. Acoramidis was well tolerated.
CONCLUSIONS: Acoramidis prolongs survival, provides functional and quality-of-life benefits, and is well tolerated in Japanese patients with ATTR-CM, consistent with results from the global ATTRibute-CM Phase 3 trial.
METHODS AND RESULTS: Twenty-five patients (mean [±SD] age 76.5±6.3 years; 88% men) received acoramidis hydrochloride (800 mg twice daily) for up to 30 months. Primary endpoints were change from baseline to Month 12 in the 6-min walk distance (6MWD) and all-cause mortality and cardiovascular-related hospitalizations over 30 months. No deaths were reported over 30 months. The annualized frequency of cardiovascular-related hospitalizations was 0.133. Clinically meaningful treatment benefits were observed in least-squares mean change from baseline to Month 12 for 6MWD (-3.9 m; 95% confidence interval [CI] -22.9, 15.1 m) and to Month 30 for 6MWD (-36.2 m; 95% CI -58.5 to -13.9 m), Kansas City Cardiomyopathy Questionnaire overall score (-7.0; 95% CI -14.7, 0.8), TTR levels (9.4 mg/dL; 95% CI 6.7, 12.0 mg/dL), and median N-terminal pro B-type natriuretic peptide levels (169.5 pg/mL; interquartile range -557.0, 700.0 pg/mL). The median TTR stabilization (fluorescent probe exclusion assay) was 95.6%. Acoramidis was well tolerated.
CONCLUSIONS: Acoramidis prolongs survival, provides functional and quality-of-life benefits, and is well tolerated in Japanese patients with ATTR-CM, consistent with results from the global ATTRibute-CM Phase 3 trial.