Phase III post-hoc analyses of Vyndaqel shows benefits in ATTR-ACT.- Pfizer.

Pfizer announced that additional sensitivity and post-hoc analyses from the Tafamidis Phase III Transthyretin Amyloid Cardiomyopathy (ATTR-ACT) study provide further detail on the effect of Vyndaqel (tafamidis) across wild-type, hereditary, and New York Heart Association (NYHA) class sub-groups of patients with transthyretin amyloid cardiomyopathy (ATTR-CM). Tafamidis is the only investigational treatment that has completed a Phase III trial evaluating its safety and efficacy for the treatment of ATTR-CM. The broader primary results showed tafamidis significantly reduced the hierarchical combination of both all-cause mortality and frequency of cardiovascular-related hospitalizations compared to placebo over a 30-month period (P=0.0006) in patients with wild-type and hereditary ATTR-CM.

A new sensitivity analysis also demonstrated a significant reduction in the combination of all-cause mortality and frequency of all-cause hospitalization compared to placebo over a 30-month period (P=0.0088). In addition, tafamidis reduced the risk of all-cause mortality across all sub-groups (wild-type, hereditary and NYHA I, II and III functional class) versus placebo. This included a 29% and 31% reduction in the risk of death observed in wild-type (HR 0.71; 95% CI [0.474, 1.052]) and hereditary (HR 0.69; 95% CI [0.408,1.167]) sub-groups, respectively. Across wild-type and hereditary sub-groups, tafamidis consistently reduced the decline in the six minute walk test distance, a measure of functional capacity, and aspects of quality of life measured by the Kansas City Cardiomyopathy Questionnaire � Overall Score, compared with placebo at Month 30.

Tafamidis was also well tolerated, with an observed safety profile comparable to placebo. The findings were presented during the Late Breaking Clinical Trials session at the Heart Failure Society of America 22nd Annual Scientific Meeting in Nashville, TN.