Pfizer Inc. (PFE: Quote) announced Thursday that the United States Food and Drug Administration's or 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 or TTR-FAP in adult patients with symptomatic polyneuropathy to delay neurologic impairment.
The company said that 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.
The company noted that the recommendation will be taken into consideration by the FDA when making its decision on Pfizer's New Drug Application or NDA for tafamidis as a treatment for TTR-FAP, a rare and fatal neurodegenerative disease.
The FDA has granted the tafamidis NDA both an orphan drug and a priority review designation.
Transthyretin Familial Amyloid Polyneuropathy is a rare and fatal neurodegenerative disease, primarily caused by a genetic mutation of the transthyretin or TTR gene.1,2 In TTR-FAP, TTR destabilization leads to misfolded proteins that form amyloid fibrils in the peripheral and autonomic nerves, as well as other organs including the GI tract, kidneys and heart.
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by RTT Staff Writer
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