FDA Advisory Committee votes on tafamidis (Pfizer) for TTR-FAP

The FDA Peripheral and Central Nervous System Drugs Advisory Committee voted on the clinical data package for tafamidis meglumine submitted by Pfizer. 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 the New Drug Application for tafamidis as a treatment for TTR-FAP, a rare and fatal neurodegenerative disease.The drug is approved in the EU for this indication.