The United States Food and Drug Administration (FDA) has granted the drug tafamidis Breakthrough Therapy designation for treating patients who have transthyretin cardiomyopathy. The full article can be read here, at Pfizer.
Transthyretin cardiomyopathy (TTR-CM) is a rare condition that is underdiagnosed in patients. It is associated with heart failure and restrictive cardiomyopathy. TTR-CM occurs when the protein transthyretin accumulates in the heart causing heart stiffness and failure. A fact sheet on the condition states that on average, people who are diagnosed with the condition will live for another three to five years. Roughly one thousand people are believed to be diagnosed with TTR-CM, but this is thought to be a significant underestimation of the actual number of people with the condition. There are two forms of the TTR-CM: wild-type TTR-CM, which is not inherited, and TTR familial amyloid cardiomyopathy that is hereditary.
There are not currently any FDA approved treatments specifically for TTR-CM that address the underlying cause of the disease. However, topline data from a Phase 3 trial of tafamidis in patients with TTR-CM suggests that tafamidis could be an effective treatment for the condition in the future. The study showed that patients taking tafamidis had a significantly lower rate of combined all-cause mortality and cardiovascular-related hospitalisation. Brenda Cooperstone MD, a senior member of Pfizer Rare Disease, says,
“This designation is an important step forward.”
