A new phase III clinical trial has demonstrated that Pfizer’s tafamidis drug can lower the risk of death in patients suffering from rare heart condition transthyretin amyloid cardiomyopathy.

Data revealed a 30% decrease in the risk of mortality and 32% reduction in the rate of cardiovascular-related hospitalisations at 30 months, compared to placebo.

Pfizer presented the results at the European Society of Cardiology’s annual conference in Germany and published in the New England Journal of Medicine (NEJM).

Conducted in a total of 441 patients, the ATTR-ACT study assessed the efficacy, safety and tolerability of oral 20mg or 80mg daily dose of tafamidis meglumine capsules.

The findings from the trial included a favourable profile with secondary study endpoints, including decrease of the decline in the six minute walk test distance, which represents functional capacity.

Pfizer’s tafamidis was also found to have minimised the decline in quality of life aspects as measured using the Kansas City Cardiomyopathy Questionnaire at month 30.

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“We believe the ATTR-ACT study findings bring us a significant step closer to our goal of providing an urgently needed therapy for a serious disease.”

During the trial, tafamidis was well-tolerated and its safety profile was comparable to that of placebo.

Pfizer Global Product Development, Rare Disease senior vice-president and chief development officer Brenda Cooperstone said: “We believe the ATTR-ACT study findings bring us a significant step closer to our goal of providing an urgently needed therapy for a serious and often fatal disease.

“We look forward to continuing discussions with global regulatory authorities about the potential of tafamidis as a treatment option for people living with ATTR-CM.”

Transthyretin amyloid cardiomyopathy is a rare, fatal and underdiagnosed disorder that can lead to progressive heart failure. The condition is said to currently lack approved pharmacologic therapies.

Pfizer’s tafamidis secured orphan drug designation for treating the rare heart condition in 2012 in both the US and European Union.

Later, the US Food and Drug Administration also granted fast track and breakthrough therapy designations. This year, the drug obtained orphan drug and SAKIGAKE designations in Japan.