Acoramidis for Transthyretin Amyloid Cardiomyopathy

Transthyretin (TTR) amyloid cardiomyopathy results from the destabilization of tetramers into monomers that accumulate in the myocardium and clinically manifests as heart failure with preserved systolic function, which can be markedly symptomatic. Prior studies show that tafamidis, a TTR stabilizer, improves outcomes for this condition. This phase 3, manufacturer-sponsored, double-blind, placebo-controlled trial (NCT03860935) evaluated acoramidis, a non–FDA-approved compound that mimics the action of a rare genetic TTR mutation with greater potency and TTR stabilization than tafamidis.

The investigators randomized 632 individuals with established TTR amyloid cardiomyopathy (median age, 77 years; 90% men; 88% white; 89% with New York Heart As…