Tafamidis is an oral, investigational, small molecule developed to treat transthyretin amyloid cardiomyopathy. Credit: Bryan Brandenburg.

The US Food and Drug Administration (FDA) has accepted Pfizer’s two new drug applications (NDAs) for tafamidis to treat transthyretin amyloid cardiomyopathy (ATTR-CM).

Tafamidis is an oral, investigational, small molecule designed for selective binding at specific sites on the transthyretin tetramer in order to prevent transthyretin transport protein destabilisation and amyloid formation that leads to ATTR-CM.

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The NDAs seek approval for meglumine salt and free acid forms of the drug.  The meglumine form has obtained priority review status from the FDA, while the free acid form will be under standard review.

Pfizer global product development rare disease senior vice-president and chief development officer Brenda Cooperstone said: “The diagnosis of ATTR-CM is often delayed, primarily because disease awareness is low and patients often present with symptoms similar to more common causes of heart failure.

“The NDAs include results from the Phase III Transthyretin Amyloid Cardiomyopathy (ATTR-ACT) clinical trial that assessed the efficacy, safety and tolerability of tafamidis meglumine.”

“The FDA’s filing acceptance is an encouraging step toward our goal of further raising awareness and providing a treatment option for ATTR-CM patients who are in desperate need of an approved pharmacologic therapy. We look forward to working with the FDA to bring the first treatment for this deadly disease to patients.”

The NDAs include results from the Phase III Transthyretin Amyloid Cardiomyopathy (ATTR-ACT) clinical trial that assessed the efficacy, safety and tolerability of tafamidis meglumine in 441 wild-type or hereditary ATTR-CM patients.

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Primary analysis of the trial showed that the drug met the primary endpoint of significant decrease in hierarchical combination of all-cause mortality and frequency of cardiovascular-related hospitalisations over 30 months, compared to placebo.

Tafamidis was found to be well tolerated, with an observed safety profile.

Tafamidis secured the FDA fast track and breakthrough therapy designations in June 2017 and May 2018 respectively.