What question did this study set out to answer?

This trial aimed to evaluate the efficacy and safety of acoramidis in treating symptomatic transthyretin amyloid cardiomyopathy in Japanese adults.

April 25, 2026Open Access

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

Puntos clave

This trial aimed to evaluate the efficacy and safety of acoramidis in treating symptomatic transthyretin amyloid cardiomyopathy in Japanese adults.
Multicenter open-label single-arm Phase 3 trial (NCT04622046)
Involved Japanese patients with wild-type or hereditary symptomatic transthyretin amyloid cardiomyopathy
Assessed pharmacokinetics and pharmacodynamics of acoramidis
Significant efficacy observed in symptomatic relief from transthyretin amyloid cardiomyopathy
Safety profile of acoramidis aligned with expectations for TTR stabilizers
Pharmacokinetic and pharmacodynamic results supported its therapeutic use

Resumen

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).

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