Pfizer Drug Reduces Mortality and Hospitalizations in Transthyretin Cardiomyopathy Patients

Positive topline results from Pfizer’s Phase 3 Transthyretin Cardiomyopathy (ATTR-ACT) study met its primary endpoint, as tafamidis exhibited a statistically significant reduction in the combination of all-cause mortality and frequency of cardiovascular-related hospitalizations compared to placebo in transthyretin cardiomyopathy patients.

The preliminary safety data showed that, at 30 months, tafamidis was generally well tolerated in this patient population, and no new safety signals were identified.

The study was designed to evaluate clinically meaningful outcomes for the use of tafamidis as a treatment for patients with transthyretin cardiomyopathy. At present, there are no approved pharmacological medications specifically indicated for treatment the progressive heart condition, and the average life expectancy of patients is 3 to 5 years after diagnosis.

In the study, 441 participants were randomized into 3-arms that investigated the efficacy, safety, and tolerability of an oral dose of either 20 mg or 80 mg tafamidis meglumine capsules compared to placebo. Patients in the third arm received placebo.

“Our findings offer real hope for people with transthyretin cardiomyopathy and their families,” said Mat Maurer MD, Arnold and Arlene Goldstein professor of Cardiology, Columbia University Vagelos College of Physicians and Surgeons. “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. The need for medicines that treat transthyretin cardiomyopathy is critical.”

“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 Brenda Cooperstone MD, senior vice president and chief development officer, Rare Disease, Pfizer Global Product Development in a press release.

In 2011, tafamidis was granted orphan drug designation for transthyretin cardiomyopathy in both the European Union (EU) and the U.S., and 6 years later in 2017, the U.S. Food and Drug Administration (FDA) granted the drug Fast Track designation for the indication.

“Pfizer Rare Disease has been at the forefront of improving the understanding of transthyretin cardiomyopathy, and we thank the patients who participated in the trial and their families, as well as the physicians and investigational sites that contributed to this important study,” Dr Cooperstown continued. “We look forward to sharing the detailed results of the study with the cardiovascular community and discussing these data with health authorities to determine an appropriate regulatory path forward.”

The results are subject to further analysis and full data will be submitted for presentation at an upcoming scientific congress.

For more on breakthrough therapies from the rare disease community, follow Rare Disease Report on Facebook and Twitter.