Efficacy and Safety of Acoramidis in Japanese Adults With Symptomatic Transthyretin Amyloid Cardiomyopathy - PubMed

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Acoramidis (800 mg twice daily) was evaluated in a Phase 3 trial involving 25 Japanese adults (mean age 76.5 years, 88% men) with symptomatic ATTR-CM.
Primary endpoints included change in 6-minute walk distance (6MWD) at 12 months and cardiovascular-related hospitalizations over 30 months; no deaths occurred, and the annualized hospitalization rate was low at 0.133.
Clinically meaningful benefits were observed over 30 months in 6MWD, quality-of-life scores, TTR levels, and NT-proBNP, with median TTR stabilization at 95.6%, and the treatment was well-tolerated.
The study concluded that acoramidis improves survival, function, and quality of life in Japanese ATTR-CM patients, aligning with global Phase 3 trial results.

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