FDA 'Breakthrough' status for tafamidis and Lenti-D

The US Food and Drug Administration yesterday announced two more drug candidates have been granted coveted Breakthrough Therapy designations (BTD).

One of these was US pharma giant Pfizer (NYSE:PFE) tafamidis 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 top-line results from the tafamidis Phase III 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 hospitalizations. Currently, there are no approved pharmacological treatments specifically indicated for this disease, and the average life expectancy for people with transthyretin cardiomyopathy is three to five years from diagnosis.