Oral Tx for Transthyretin Cardiomyopathy Meets Primary Endpoint in Study

Patients were randomized to oral tafamidis meglumine 20mg or 80mg, or placebo
Patients were randomized to oral tafamidis meglumine 20mg or 80mg, or placebo

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.