Inaugural Treatment for Transthyretin Amyloid Cardiomyopathy

A disheartening cardiovascular disorder, ATTR-CM is a progressive, underdiagnosed, potentially fatal disease in which amyloid protein fibrils deposit in, and stiffen, the walls of the heart’s left ventricle. But a new agent to prevent misfolding of the deposited protein is showing a significantly reduced risk of death. Following Fast-Track and Breakthrough designations in 2017 and 2018, 2019 marked the FDA approval of tafamidis, the first-ever medication for treatment of this increasingly recognized condition.