Rare Daily Staff

Pfizer said that its drug tafamidis met its primary endpoint in a late-stage study in patients with transthyretin cardiomyopathy, a rare, fatal, and underdiagnosed condition associated with progressive heart failure.

The average life expectancy for people with transthyretin cardiomyopathy is three to five years from diagnosis. The prevalence of transthyretin cardiomyopathy is presently unknown, but it is believed that less than 1 percent of people with the disease are diagnosed. Currently, there are no drugs specifically indicated for treating transthyretin cardiomyopathy.

The study found a statistically significant reduction in the combination of all-cause mortality and frequency of cardiovascular-related hospitalizations compared to placebo at 30 months. The preliminary safety data showed that tafamidis was generally well tolerated in this population and no new safety signals were identified.

“As health care professionals, all we can do right now is manage symptoms of the disease, as there are no approved pharmacological treatment options at this time,” said Mat Maurer professor of Cardiology, Columbia University Vagelos College of Physicians and Surgeons. “The need for medicines that treat transthyretin cardiomyopathy is critical.”

In 2011, tafamidis was granted orphan drug designation for transthyretin cardiomyopathy in both the United States and European Union. In June 2017, the U.S. Food and Drug Administration 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.

March 30, 2018
Photo: Mat Maurer professor of Cardiology, Columbia University Vagelos College of Physicians and Surgeons