Heart HealthPhase 3 Trial Tests Tafamidis for TTR Amyloid Heart Disease
Transthyretin amyloid cardiomyopathy (ATTR-CM) occurs when misfolded transthyretin protein deposits accumulate in heart tissue, leading to progressive heart failure. Tafamidis is a small molecule that stabilizes the transthyretin protein tetramer, preventing it from breaking apart and forming damaging amyloid fibrils. This Pfizer-sponsored Phase 3 trial enrolled 441 patients with either hereditary or wild-type ATTR-CM and randomly assigned them to receive 20 mg tafamidis, 80 mg tafamidis, or placebo daily for approximately four years. The trial aimed to establish whether tafamidis could reduce mortality, hospitalizations, and functional decline compared to placebo. Completed in 2018, this study served as the pivotal evidence base for regulatory review of tafamidis in heart failure caused by TTR amyloidosis.
