ATTRibute-CM

Efficacy and Safety of Acoramidis (AG10) in Subjects With Transthyretin Amyloid Cardiomyopathy

Patient / Population Intervention / Exposure Comparison Outcome

In 632 patients with transthyretin amyloid cardiomyopathy, acoramidis improved a hierarchical composite of death, CV hospitalisation, NT-proBNP and 6-minute walk distance compared with placebo.

632

Design

Double-blind placebo-controlled RCT, phase 3

Endpoint

Hierarchical composite (all-cause death, CV hospitalisation, NT-proBNP, 6-minute walk) at 30 months

Relevance

ResultAcoramidis superior on the hierarchical primary endpoint (win ratio 1.8, 95% CI 1.4-2.2; P<0.001), driven largely by mortality and CV hospitalisation.

Gillmore JD, et al. N Engl J Med. 2024;390(2):132-142. 10.1056/NEJMoa2305434

Discussion & critique

Win-ratio composite harder to interpret clinically than event rates, and high background tafamidis use complicates comparison. Expanded disease-modifying options for ATTR-CM as a second-generation TTR stabiliser.