New long-term data published in JAMA Cardiology confirms that acoramidis, an approved therapy for transthyretin amyloid cardiomyopathy, delivers sustained and durable benefits over 54 months,  cutting the risk of cardiovascular mortality by 49 percent and all-cause mortality by 45 percent compared to placebo. The findings, drawn from an open-label extension of the landmark ATTRibute-CM randomized clinical trial, represent the longest efficacy and safety data yet reported for this class of treatment.

Transthyretin amyloid cardiomyopathy is caused by the misfolding and accumulation of the transthyretin protein in heart tissue, progressively impairing cardiac function. The condition is currently classified and treated as a rare disease, yet post-mortem studies suggest it is substantially more prevalent than diagnosed, with a significant proportion of very elderly individuals showing potentially life-threatening levels of transthyretin accumulation in the heart and other organs. The gap between clinical recognition and actual disease burden reflects both the subtlety of early symptoms and the high cost of treatment, which is priced for rare-disease markets.

The extension study enrolled 389 participants from the original 632-person trial, with a mean age of 77 years. Those who received continuous acoramidis from the outset showed consistent reductions in cardiovascular hospitalization risk (hazard ratio 0.53) through month 54, alongside stabilized heart failure symptoms, preserved walking capacity, and controlled levels of NT-proBNP, a key blood marker of cardiac stress. Participants who had been on placebo and switched to acoramidis at month 30 also showed meaningful stabilization and improvement across multiple measures by month 54. No new long-term safety concerns were identified.

The broader significance of these results extends beyond the currently diagnosed patient population. Transthyretin amyloidosis functions as a universal mechanism of aging, one that operates to some degree in virtually all older individuals, not only those with genetic mutations or severe symptomatic disease. As these drugs approach generic availability and treatment costs eventually fall, the accumulating long-term data will be critical in making the scientific and policy case for wider use. The 54-month ATTRibute-CM findings add meaningfully to that evidence base.