Pfizer Inc. said its drug tafamidis received the U.S. Food and Drug Administration's breakthrough therapy designation for treating a condition linked to progressive heart failure.
Tafamidis is an investigational therapy for transthyretin cardiomyopathy, a rare, fatal and underdiagnosed condition associated with progressive heart failure. Transthyretin cardiomyopathy is a form of transthyretin amyloidosis, or ATTR, a progressive condition caused by mutations in the transthyretin gene.
The New York-based pharmaceutical company said the decision is supported by a phase 3 study in which tafamidis reduced premature deaths and frequency of heart-related hospitalizations.
There are no approved treatments for transthyretin cardiomyopathy. People with this condition have an average life expectancy of three to five years as of diagnosis.
Tafamidis received orphan drug designation for the disease in both the EU and the U.S. in 2012. The FDA granted fast-track designation to the experimental drug for the same use in 2017.
Meanwhile, Japan's Ministry of Health, Labour and Welfare granted tafamidis a Sakigake designation — similar to the FDA's breakthrough therapy designation — for the disease in 2018.
