Pfizer has reported positive topline results from the Transthyretin Cardiomyopathy (ATTR-ACT) study, evaluating tafamidis for the treatment of transthyretin cardiomyopathy.

The Phase III trial has met its primary endpoint, achieving a statistically significant reduction in the combination of all-cause mortality and frequency of cardiovascular-related hospitalisations compared to placebo at 30 months.

Preliminary safety data of the trial demonstrated that tafamidis was generally well tolerated in this population, with no new safety signals identified.

“These topline results bring us one step closer to realising the potential for a new treatment for those in desperate need.”

The international, three-arm ATTR-ACT clinical trial enrolled 441 patients.

The efficacy, safety, and tolerability of an oral daily dose of 20mg or 80mg tafamidis meglumine capsules was compared to placebo.

Patients with both the variant – or hereditary – form of the disease and those with the wild-type form took part in the trial. The latter type is not hereditary and may occur as people age.

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Pfizer Rare Disease Global Product Development senior vice-president and chief development officer Brenda Cooperstone said: “These topline results are important for people with transthyretin cardiomyopathy and bring us one step closer to realising 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.”

Transthyretin cardiomyopathy is a rare, fatal, and underdiagnosed condition associated with progressive heart failure.

The average life expectancy for people suffering from transthyretin cardiomyopathy is three to five years from diagnosis.

Currently, there are no approved pharmacological medications available for specifically treating transthyretin cardiomyopathy.