Pfizer Inc. announced that tafamidis received Breakthrough Therapy designation from the US Food and Drug Administration (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 hospitalisation. 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.”

Breakthrough Therapy designation was initiated as part of the Food and Drug Administration Safety and Innovation Act (FDASIA) signed in 2012. As defined by the FDA, a breakthrough therapy is a drug intended to be used alone or in combination with one or more other drugs to treat a serious or life-threatening disease or condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development. If a drug is designated as a breakthrough therapy, the FDA may expedite the development and review of such drug.

Tafamidis is an investigational treatment for transthyretin cardiomyopathy and is not approved for this indication. In 2012, tafamidis was granted orphan drug designation for transthyretin cardiomyopathy in both the EU and US. In May 2017, the US FDA granted Fast Track designation to tafamidis for transthyretin cardiomyopathy; additionally, in March 2018, the Ministry of Labor Health and Welfare in Japan granted SAKIGAKE designation to tafamidis for this indication.

Transthyretin cardiomyopathy is a rare, fatal, and underdiagnosed condition associated with progressive heart failure. The prevalence of transthyretin cardiomyopathy is presently unknown; however, it is estimated that less than 1% of people with the disease are diagnosed. Currently, there are no approved pharmacological treatments specifically indicated for this disease, and the average life expectancy is 3 to 5 years from diagnosis.

Rare disease includes some of the most serious of all illnesses and impacts millions of patients worldwide,6 representing an opportunity to apply our knowledge and expertise to help make a significant impact on addressing unmet medical needs. The Pfizer focus on rare disease builds on more than two decades of experience, a dedicated research unit focusing on rare disease, and a global portfolio of multiple medicines within a number of disease areas of focus, including hematology, neuromuscular, and inherited metabolic disorders.

Pfizer Rare Disease combines pioneering science and deep understanding of how diseases work with insights from innovative strategic collaborations with academic researchers, patients, and other companies to deliver transformative treatments and solutions. We innovate every day leveraging our global footprint to accelerate the development and delivery of groundbreaking medicines and the hope of cures.