A phase III clinical trial showed that a combination of 80 mg of tafamidis meglumine and 61 mg of tafamidis performed better than those receiving 20 mg of tafamidis meglumine for patients with transthyretin amyloid cardiomyopathy (ATTR-CM).
A press release from the manufacturer noted that in the ATR-ACT trial and extension study, patients were initially treated with the 80 mg drug before transitioning to the 61 mg dose, while a second group started with the 20 mg drug before transitioning to the 61 mg treatment.
The release noted that after adjusting for several factors, including age, biomarkers, and functional capacity, there was a 43% reduction in the risk of death in the first group.
“Results from this analysis provide valuable information for health care providers by reinforcing that VYNDAQEL 80 mg and the bioequivalent tafamidis free acid 61 mg VYNDAMAX are the appropriate doses for patients with ATTR-CM, demonstrating a clear survival benefit of VYNDAQEL 20 mg,” said Brenda Cooperstone, Chief Developmental Officer, Rare Disease, Pfizer Global Product Development.
The release describes ATTR-CM as a “rare, undiagnosed, progressive and life-threatening disease,” which can cause heart muscles to stiffen, eventually leading to heart failure. The median life expectancy, depending on sub-type, is up to 3.5 years.
The long-term extension combined with the data from the ATTR-ACT median follow-up was 51 months.
Other findings from the study included a relative reduction of 30% in the risk of all-cause mortality and a 32% reduction in cardiovascular-related hospitalization frequency between the drug and placebo groups.
“With additional patients and a longer duration of treatment, data from the extension study provided further confirmation that VYNDAQEL 80mg and VYNDAMAX 61mg are the optimal doses for the treatment of ATTR-CM,” said Thibaud Damy, MD, from the French Society of Cardiology, in the release. “These medicines, which provide a significant survival benefit, represent a breakthrough for patients with ATTR-CM who previously had limited treatment options.”
The release noted that the safety profile of all three drugs was similar to placebo.
