Success for Phase III study of tafamidis meglumine for the treatment of transthyretin cardiomyopathy.- Pfizer.

Pfizer Inc.has announced that the Tafamidis Phase III Transthyretin Cardiomyopathy (ATTR-ACT) study evaluating tafamidis for the treatment of transthyretin cardiomyopathy met its primary endpoint, demonstrating a statistically significant reduction in the combination of all-cause mortality and frequency of cardiovascular-related hospitalizations compared to placebo at 30 months. The preliminary safety data showed that tafamidis was generally well tolerated in this population and no new safety signals were identified.

The ATTR-ACT study was designed to assess clinically meaningful outcomes for the use of tafamidis as a treatment for patients with transthyretin cardiomyopathy, a rare, fatal, and underdiagnosed condition associated with progressive heart failure. The average life expectancy for people with transthyretin cardiomyopathy is 3 to 5 years from diagnosis. The prevalence of transthyretin cardiomyopathy is presently unknown; however, it is estimated that less than 1% of people with the disease are diagnosed. Currently, there are no approved pharmacological medications specifically indicated for treating transthyretin cardiomyopathy.