On Thursday, March 29th, Pfizer announced positive topline results from ATTR-ACT trial of tafamidis in patients with transthyretin cardiomyopathy. According to Pfizer, “Tafamidis met its primary endpoint, demonstrating a statistically significant reduction in the composite 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”.
ATTR-ACT is a multicenter, international, phase 3, double-blinded, placebo-controlled trial which randomized 441 patients to placebo or one of two oral doses of the drug (20 and 80 mg daily). This study was designed to assess clinically meaningful outcomes (efficacy, safety, and tolerability) for the use of tafamidis meglumine as a treatment for patients with transthyretin cardiomyopathy (ttr-cm), a rare, fatal, and underdiagnosed condition associated with progressive heart failure.
“Currently, there are no approved pharmacological medications specifically indicated for treating transthyretin cardiomyopathy”, as mentioned by the drug company in Pfizer’s press release. The average life expectancy for people with transthyretin cardiomyopathy is 3 to 5 years from diagnosis. “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 of Pfizer Global Product Development.
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.
Dr. Mathew Maurer, Cardiology Professor at Columbia University commented; “Our findings offer real hope for people with transthyretin cardiomyopathy and their families. 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.”