CHMP recommends approval of Vyndaqel in hereditary transthyretin amyloidosis.- Pfizer

Pfizer announced that the Committee for Medicinal Products for Human Use of the European Medicines Agency (EMA) has adopted a positive opinion recommending the approval of 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).

The European line extension application was based on the Phase III ATTR-ACT study, the first and only completed global, double-blind, randomised, placebo-controlled clinical trial to investigate a pharmacologic therapy for the treatment of ATTR-CM.5 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).5

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% (p=0.026) and 32% (p<0.0001), respectively with vyndaqel versus placebo.5 the application is also based on findings from an evaluation of the free acid form of tafamidis 61 mg.6 the attr-act primary results were presented in a hot line session at the esc congress 2018 in munich germany and simultaneously published online in the new england journal of medicine nejm in august 2018.>