Pfizer’s tafamidis meets primary endpoint in phase 3 transthyretin cardiomyopathy study

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

The ATTR-ACT study was designed to assess clinically meaningful outcomes for the use of tafamidis as a treatment for patients with transthyretin cardiomyopathy, a rare, fatal, and underdiagnosed condition associated with progressive heart failure.^1,2 The average life expectancy for people with transthyretin cardiomyopathy is 3 to 5 years from diagnosis.³

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 medications specifically indicated for treating transthyretin cardiomyopathy.⁴

Pfizer Global Product Development rare disease chief development officer and senior vice president Dr Brenda Cooperstone said: “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.

“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.”

In 2011, tafamidis was granted orphan drug designation for transthyretin cardiomyopathy in both the EU and US. In June 2017, the US Food and Drug Administration (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.

ATTR-ACT is a Phase 3 international, multicenter, double-blind, placebo-controlled, randomized, 3-arm clinical study in 441 patients that investigated the efficacy, safety, and tolerability of an oral daily dose of 20 mg or 80 mg tafamidis meglumine capsules compared to placebo.

The study included both patients with the variant, or hereditary, form of the disease, and those with the wild-type form, which is not hereditary and may occur as people age.

 The primary analysis of the study, which compared tafamidis to placebo, was the hierarchical combination of all-cause mortality and frequency of cardiovascular-related hospitalizations over a 30-month period in patients with transthyretin cardiomyopathy.

Source: Company Press Release