A plain language review of the ATTRibute-CM study: efficacy and safety of acoramidis in transthyretin amyloid cardiomyopathy

<b>What is this plain language review about?</b> This plain language review describes the results of a study called ATTRibute-CM. This study looked at how well a drug called acoramidis worked to treat people with transthyretin amyloid cardiomyopathy (ATTR-CM). ATTR-CM is a type of heart disease that occurs when a protein called transthyretin (TTR) breaks down, changes shape (misfolds), and forms harmful amyloid fibrils (clumps of misfolded proteins). These amyloid fibrils then accumulate in the heart muscle, leading to thickening and stiffening of the heart walls, thereby reducing its ability to pump blood effectively. Symptoms of ATTR-CM include shortness of breath, swelling in the lower legs, and irregular heart beat. <b>What is ATTRibute-CM?</b> ATTRibute-CM is a study that recruited 632 participants with ATTR-CM who either took acoramidis HCl (the salt form of acoramidis) 800 mg or a placebo (a pill that looked like acoramidis, but did not contain any medicine) twice a day by mouth for 30 months. <b>What were the results and what do they mean?</b> Compared to participants in the placebo group, participants in the acoramidis group had better results on efficacy measures (measures of how well a treatment works) that included death due to any cause, being admitted to a hospital for a heart problem, change in NT-proBNP level (a type of protein that indicates worsening heart failure), and change in 6-minute walking distance. Common side effects included diarrhea and gout. Acoramidis showed clinical benefit in people with ATTR-CM compared with placebo. <b>Clinical trial number</b>: NCT03860935