Transthyretin amyloid cardiomyopathy (ATTR-CM), caused by destabilization of serum transthyretin (sTTR), is a progressive and life-threatening disorder associated with worsening cardiac function and limited long-term therapeutic data despite advances in transthyretin stabilizing therapies. To address this gap, the ATTRibute-CM study and its open-label extension evaluated the long-term efficacy and safety of acoramidis, a therapy achieving near-complete (≥90%) sTTR stabilization, with assessment of sustained clinical outcomes through month 54 in 632 participants with ATTR-CM.

The study found that continuous acoramidis treatment through month 54 significantly reduced the risks of all-cause mortality (HR, 0.55), cardiovascular mortality (HR, 0.51), and first cardiovascular hospitalization (HR, 0.53) compared with delayed treatment initiation. Participants receiving continuous acoramidis…