Pfizer announced that the Food and Drug Administration (FDA) has accepted the New Drug Applications (NDAs) for tafamidis meglumine and tafamidis free acid for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM). 

Tafamidis is an oral small molecule candidate that selectively binds at specific sites on the transthyretin tetramer to prevent destabilization of the transthyretin transport protein and formation of amyloid that causes ATTR-CM. The tafamidis meglumine formulation (20mg capsules) has been granted Priority Review, while the free acid formulation (61mg capsules [bioequivalent to 80mg tafamidis meglumine]) will undergo Standard Review; the free acid formulation was developed for patient convenience, allowing for daily administration of a single capsule.

The NDA submissions were supported by data from the Phase 3 multicenter, double-blind, placebo-controlled, randomized, 3-arm Transthyretin Amyloid Cardiomyopathy (ATTR-ACT) study which compared tafamidis meglumine (20mg or 80mg) vs placebo in 441 patients. Treatment with tafamidis met the primary endpoint in the primary analysis, demonstrating a significant reduction in the combination of all-cause mortality and frequency of cardiovascular-related hospitalizations vs placebo at 30 months in patients with wild-type or hereditary ATTR-CM (P =.0006). 

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The FDA has set a target Prescription Drug User Fee Act (PDUFA) date of July 2019 for tafamidis meglumine and November 2019 for tafamidis free acid. 

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