EU approves Vyndaqel for hereditary transthyretin amyloidosis with cardiomyopathy.- Pfizer

Pfizer announced that the European Commission (EC) has approved Vyndaqel (tafamidis), a once-daily 61 mg oral capsule, for the treatment of wild-type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM). Vyndaqel is the first and only treatment approved in the European Union for patients with ATTR-CM. Prior to this approval, treatment options for patients with ATTR-CM were restricted to symptom management, and, in rare cases, heart (or heart and liver) transplant.The EC approval of Vyndaqel is based on results from the Phase III ATTR-ACT study, the first and only completed global, double-blind, randomized, placebo-controlled clinical trial to investigate a pharmacologic therapy for the treatment of ATTR-CM. The study compared patients who received an oral daily dose of 20 mg or 80 mg of tafamidis meglumine compared to those who received placebo. In the primary analysis of the study, Vyndaqel (tafamidis meglumine) demonstrated a significant reduction in the hierarchical combination of all-cause mortality and frequency of cardiovascular-related hospitalisations compared to placebo over a 30-month period in patients with wild-type or hereditary ATTR-CM (p=0.0006). Additionally, individual components of the primary analysis demonstrated a relative reduction in the risk of all-cause mortality and frequency of cardiovascular-related hospitalization of 30% and 32%, respectively, with Vyndaqel versus placebo. Vyndaqel also had significant and consistent treatment effects compared to placebo on functional capacity and health status first observed at six months and continuing through 30 months. Specifically, Vyndaqel reduced the decline in performance on the six-minute walk test and reduced the decline in health status as measured by the Kansas City Cardiomyopathy Questionnaire – Overall Summary score. Vyndaqel was well tolerated in this study, with an observed safety profile comparable to placebo.