FDA advisory panel gives mixed views on Pfizer's tafamidis

The US Food and Drug Administration's peripheral and Central Nervous System Drugs Advisory Committee yesterday voted on pharma behemoth Pfizer's (NYSE: PFE) clinical data package for tafamidis meglumine expressing mixed views on the data submitted, with the majority of panelists agreeing there was sufficient evidence of its effectiveness to meet FDA criteria for an accelerated approval of a drug.

Tafamidis is a novel, investigational, oral therapy for the treatment of Transthyretin Familial Amyloid Polyneuropathy (TTR-FAP) in adult patients with symptomatic polyneuropathy to delay neurologic impairment, a rare, progressive and fatal hereditary neurodegenerative disease that affects around 8,000 patients worldwide.

The panel of experts did not find substantial evidence of efficacy on a clinical endpoint. The Committee then voted 13 to four that the data backing Pfizer’s New Drug Application provide substantial evidence of efficacy for a surrogate endpoint that is reasonably likely to predict a clinical benefit. This recommendation will be taken into consideration by the FDA when making its decision on Pfizer's NDA for tafamidis as a treatment for TTR-FAP, a rare and fatal neurodegenerative disease.