FDA advisory committee votes in favor of Pfizer's tafamidis meglumine

May 25 2012

Pfizer Inc. (NYSE:PFE) announced today that the United States Food and Drug Administration's (FDA) Peripheral and Central Nervous System Drugs Advisory Committee voted on Pfizer's clinical data package for tafamidis meglumine. 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. The Advisory Committee did not find substantial evidence of efficacy on a clinical endpoint. The Committee then voted 13-4 that the data 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 New Drug Application (NDA) for tafamidis as a treatment for TTR-FAP, a rare and fatal neurodegenerative disease.    

"TTR-FAP is an irreversible and devastating disease with no FDA-approved treatment in the U.S.," said Dr. Yvonne Greenstreet, senior vice president and head of Medicines Development Group for Pfizer's Specialty Care Business Unit. "The panel's assessment represents a positive step forward in our goal to provide this much-needed medicine to patients suffering from this rare and fatal disease. Pfizer will continue to work with the FDA as the Agency finalizes its review of our NDA for tafamidis."