Tafamidis accepted for FDA Review in 2019

Earlier this month, Pfizer announced that it has made two separate FDA submissions for their drug tafamidis, based on evidence generated from the Phase 3 Transthyretin Amyloid Cardiomyopathy (ATTR-ACT) clinical trial,

This trial evaluated the efficacy, safety, and tolerability of tafamidis meglumine compared to placebo for the treatment of transthyretin amyloidosis (ATTR) with cardiomyopathy (ATTR-CM). Both submissions have been accepted for FDA review.

The two submissions are for different formulations of the drug tafamidis. The first is a 20 mg tafamidis meglumine capsule. This was the formulation of the drug used during the ATTR-ACT trial and can be given in either 20 mg (1 capsule daily) or 80 mg (4 capsules daily) doses. This submission was granted Priority Review by the FDA and has a target decision date of July 2019.

The second submission was for a free acid form of tafamidis. The free acid form has the same effect as 80 mg (or 4 capsules) of tafamidis meglumine. This form was made for the convenience of patients that may be prescribed an 80 mg dose of tafamidis. Both the 20 mg tafamidis meglumine and free acid form of tafamidis have the same mechanism of action and biologic effect, but simply provide different doses. The submission for the free acid form of tafamidis was accepted under Standard Review by the FDA and has a target decision date of November 2019.

Tafamidis has already been approved in many countries for the treatment of hereditary ATTR polyneuropathy, but has not been approved by the FDA in US. If this submission is approved, tafamidis would be the first drug approved for use in ATTR patients with cardiomyopathy (heart involvement), including both patients with wildtype ATTR and hereditary ATTR.

To read more about the tafamidis FDA submission click here >>