Rare Disease Report

Tafamidis Gets Breakthrough Therapy Designation

MAY 24, 2018
Mathew Shanley
This morning, Pfizer announced that its investigational therapy for the treatment of patients with transthyretin (TTR) cardiomyopathy, tafamidis, received Breakthrough Therapy designation from the US Food and Drug Administration (FDA).

The FDA’s decision is supported by topline results from the phase 3 Transthyretin Cardiomyopathy (ATTR-ACT) study in which tafamidis exhibited a statistically significant reduction in the combination of all-cause mortality and frequency of cardiovascular-related hospitalizations. The positive topline results were released in March.

TTR is a tetramer that is responsible in transporting the retinol-binding protein-vitamin A complex and minimally transporting thyroxine in the blood. Tafamidis is also being developed as a novel specific TTR stabilizer or dissociation inhibitor.

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 of tafamidis meglumine capsules or placebo. The preliminary safety data showed that, at 30 months, tafamidis was generally well tolerated in the patient population, and no new safety signals were identified.

“This designation is an important step forward in the path to bringing a potential new treatment option to those with transthyretin cardiomyopathy, a rare, fatal disease,” said Brenda Cooperstone MD, Senior Vice President and Chief Development Officer, Rare Disease, Pfizer Global Product Development in a press release. “We look forward to working with the FDA through this expedited process to fulfill an unmet patient need.”

In 2011, tafamidis was granted orphan drug designation for transthyretin cardiomyopathy in both the European Union (EU) and the US. Six years later in 2017, the FDA also granted the drug Fast Track designation for the indication.

Transthyretin cardiomyopathy is a rare, fatal, and underdiagnosed condition associated with progressive heart failure. The prevalence of transthyretin cardiomyopathy is presently unknown; however, approximately less than 1% of people with the disease receive a diagnosis.

Currently, there are no approved pharmacological medications specifically indicated for treatment of the progressive heart condition, and the average life expectancy of patients is 3 to 5 years after diagnosis.

“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,” said Mat Maurer MD, Arnold and Arlene Goldstein professor of Cardiology, Columbia University Vagelos College of Physicians and Surgeons.

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