Drug meets its primary endpoint in a phase III study for the treatment of transthyretin cardiomyopathy.

Transthyretin cardiomyopathy (TTR-CM) is a progressive, fatal disease caused by the accumulation of misfolded transthyretin (TTR) amyloid fibrils in the heart. Patients experience progressive heart failure and typically live just three to five years following diagnosis. It is estimated that 800-1000 patients have been diagnosed with TTR-CM worldwide, but this number is thought to be quite low due to difficulty with diagnosing the disease. Less than 1% of people with the disease are thought to be diagnosed. There are currently no approved treatments. 

Pfizer is developing a drug for the treatment of TTR-CM that is showing promise in the clinic. Most recently, Tafamidis met its primary endpoint in a phase 3 transthyretin cardiomyopathy (ATTR-ACT) study: a statistically significant reduction in the combination of all-cause mortality and frequency of cardiovascular-related hospitalizations compared to placebo at 30 months. The drug was also well tolerated with no new safety issues identified.

“These topline results are important for people with transthyretin cardiomyopathy and bring us one step closer to realizing the potential for a new treatment for those in desperate need,” said Pfizer Global Product Development, Rare Disease Chief Development Officer and Senior Vice President Brenda Cooperstone.

Tafamidis was designated as an orphan drug in 2011 in both the US and EU. It received a Fast Track Designation from the US Food and Drug Administration in 2017 and SAKIGAKE designation from the Ministry of Labor Health and Welfare in Japan in March 2018, both for TTR-CM.