Plain language summary of how acoramidis affects the total number of heart problems over time in people with ATTR-CM: findings from the ATTRibute-CM study

Abstract

Plain Language SummaryWhat is this summary about?Transthyretin amyloid cardiomyopathy (ATTR-CM) happens when transthyretin (TTR) protein breaks apart, forming clumps that build up in the heart, making it harder to pump blood.People with ATTR-CM often go to the hospital multiple times due to heart problems and may die from heart-related causes. Repeated hospital visits negatively affect patients and increase medical costs. Examining recurrent (events occurring more than once) and cumulative (total events over time) heart-related events (hospitalizations/death) gives an overview of disease impact.Acoramidis, a medicine taken by mouth, binds tightly to TTR, preventing it from breaking apart and forming clumps.This summary describes acoramidis’ effects on recurrent and cumulative heart-related events in people with ATTR-CM.What is ATTRibute-CM?ATTRibute-CM is a study in which participants with ATTR-CM took acoramidis or placebo (non-medicine pill) for 30 months. After 30 months, everyone who joined the open-label extension took acoramidis.What were the results?Acoramidis reduced recurrent hospitalization or death by 49% versus placebo. Many heart-related events (19% of them) occurred early, within the first 6 months. The benefits of acoramidis versus placebo increased over time, with results suggesting that acoramidis started working within the first month of treatment. In the open-label extension, participants taking acoramidis from the start were 45% less likely to have heart-related deaths than those switching from placebo to acoramidis.Results suggest acoramidis can reduce the cumulative burden of repeated hospital visits and heart-related deaths in people with ATTR-CM, with benefits suggesting an early effect within 1 month.