The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending the approval of Pfizer’s 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).
ATTR-CM is a rare, life-threatening disease characterised by the buildup of abnormal deposits of misfolded protein called amyloid in the heart and is defined by restrictive cardiomyopathy and progressive heart failure. On average, patients live only 2 to 3.5 years following diagnosis.
The European line extension application was based on the Phase 3 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.
In the primary analysis of the study, 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 hospitalisation of 30% (p=0.026) and 32% (p<0.0001), respectively, with tafamidis versus placebo, with a relative risk ratio of 0.68 (0.48 per year vs. 0.70 per year;95% CI, 0.56 to 0.81).
The application is also based on findings from an evaluation of the free acid form of tafamidis 61 mg. 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.
The CHMP’s opinion will now be reviewed by the European Commission and a final decision is expected in the coming months.