23 May 2018 - Pfizer announced today that tafamidis received breakthrough therapy designation from the US FDA for the treatment of patients with transthyretin cardiomyopathy, a rare, fatal, and under-diagnosed condition associated with progressive heart failure.
This decision is supported by topline results from the tafamidis Phase 3 Transthyretin Cardiomyopathy (ATTR-ACT) study, in which tafamidis demonstrated a statistically significant reduction in the combination of all-cause mortality and frequency of cardiovascular-related hospitalisations
Currently, there are no approved pharmacological treatments specifically indicated for this disease, and the average life expectancy for people with transthyretin cardiomyopathy is 3 to 5 years from diagnosis.