Global drug giant Pfizer recently announced that tafamidis, an investigational treatment for transthyretin cardiomyopathy received Breakthrough Therapy designation from the US Food and Drug Administration (FDA). Transthyretin cardiomyopathy is a rare, fatal, and underdiagnosed 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 hospitalizations. 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.
“This designation is an important step forward in the path to bringing a potential new treatment option to those with transthyretin cardiomyopathy, a rare, fatal disease,” said Brenda Cooperstone MD, Senior Vice President and Chief Development Officer, Rare Disease, Pfizer Global Product Development. “We look forward to working with the FDA through this expedited process to fulfill an unmet patient need.”
