The US FDA approved tafamidis meglumine (Vyndaqel) and tafamidis (Vyndamax) for the treatment of wild-type or hereditary transthyretin amyloid (ATTR) cardiomyopathy based on a trial that showed a significant reduction in mortality, cardiovascular hospitalizations, and in decline in functional capacity and quality of life. The multicenter, international, double-blind, placebo-controlled, phase 3 trial, randomly assigned 441 patients with ATTR cardiomyopathy in a 2:1:2 ratio to receive 80 mg of tafamidis, 20 mg of tafamidis, or placebo for 30 months. Tafamidis was associated with lower all-cause mortality than placebo (29.5% vs. 42.9%; hazard ratio, 0.70) and a lower rate of cardiovascular-related hospitalizations, with a relative risk ratio of 0.68 (0.48 vs. 0.70 per year). At month 30, tafamidis was also associated with a lower rate of decline in distance for the 6-minute walk test and in quality of life score. The incidence and types of adverse events were similar in the tafamidis and placebo groups. The findings indicate that tafamidis is an effective therapy for patients with ATTR cardiomyopathy. ATTR cardiomyopathy is a life-threatening disease characterized by the accumulation of amyloid fibrils composed of misfolded transthyretin protein in the heart with symptoms predominately manifested in male patients 60 years of age or older. Source: https://www.fda.gov/; https://www.nejm.org/
