Effect of acoramidis on risk of death and heart problem-related hospitalizations in people with transthyretin amyloid cardiomyopathy: a plain language summary

Plain Language SummaryWhat is this summary about?Transthyretin amyloid cardiomyopathy (ATTR-CM for short) is a serious heart condition caused when a protein called transthyretin (TTR for short) becomes unstable. In this disease, unstable TTR protein breaks apart and misfolds to form harmful clumps called amyloid fibrils, which build up in the heart over time, causing thickening of the heart muscle. This makes it difficult for the heart to pump blood effectively, resulting in heart failure. There are two main types of ATTR-CM: wild-type ATTR-CM (caused by unknown reasons) and variant ATTR-CM (caused by specific protein-destabilizing variants in the TTR gene). Medicines for ATTR-CM help people with the disease live longer and stay out of hospital. Acoramidis is a medicine available in the US, Europe, and a few other countries for treating ATTR-CM in adults.This is a summary of an investigation of participants in a clinical trial called ATTRibute-CM, in which researchers looked at the effects of acoramidis in reducing the risk of death from any cause or hospitalizations due to heart problems compared with placebo (which is an inactive treatment).What are the key takeways?People who took acoramidis had a lower combined risk of dying from any cause or of being hospitalized due to heart problems. These benefits started as early as 3 months after starting treatment with acoramidis and continued through 30 months of the study.The treatment worked consistently well across different groups of people, including men and women, people with different causes of ATTR-CM (wild-type and variant), and participants from different study countries. No major safety concerns were reported with acoramidis.The results of this study may provide useful information about the use of acoramidis for the treatment of people with ATTR-CM.Clinical trial number: NCT03860935