ransthyretin (TTR) amyloid cardiomyopathy (ATTR-CM) is a progressive, often fatal disease. TTR stabilizers bind directly to TTR, inhibiting tetramer dissociation and the resulting amyloidogenic process. This comprehensive review synthesizes clinical outcomes data from the ATTRibute-CM study program, including primary analyses, prespecified sensitivity studies, and open-label extension (OLE) follow-up, to characterize the clinical profile of acoramidis, an oral TTR stabilizer approved for ATTR-CM treatment. In clinical trials, acoramidis demonstrated consistent clinical benefits, with statistically significant reductions in the composite of all-cause mortality or first cardiovascular-related hospitalization evident within 3 months and sustained through 30 months.
