Pfizer announced topline results from the Phase 3 trial (ATTR-ACT) of tafamidis for the treatment of transthyretin cardiomyopathy. The results showed that the primary endpoint was met as evident by a statistically significant reduction in the combination of all-cause mortality and incidence of cardiovascular-related hospitalizations with tafamidis vs placebo.

A total of 441 patients were enrolled in the multicenter, double-blind, placebo-controlled, randomized ATTR-ACT study; both variant (hereditary) and wild-type form (non-hereditary) form of transthyretin cardiomyopathy were included in the study. Patients were required to have amyloid deposits in biopsy tissue and a variant TTR genotype and/or TTR precursor protein identification by immunohistochemistry, scintigraphy or mass spectrometry.

Patients were randomized into 1 of 3 arms: daily tafamidis meglumine 20mg or 80mg capsules, or placebo. The primary outcome was all-cause mortality and frequency of cardiovascular-related hospitalization, from baseline to Month 30. 

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Although the preliminary data are subject to further analysis, study authors observed a statistically significant reduction in the combination of all-cause mortality and frequency of cardiovascular-related hospitalizations compared to placebo at 30 months. Additionally, safety data showed that tafamidis was generally well-tolerated with no new safety signals.

Transthyretin cardiomyopathy is a rare and underdiagnosed condition with no approved treatments. Tafamidis was previously granted Fast Track designation for transthyretin cardiomyopathy in 2017. Full ATTR-actresults will be submitted for presentation at an upcoming scientific conference and for peer-review publication. 

For more information visit Pfizer.com.