NEW YORK — The Food and Drug Administration has declined to approve a drug designed to treat a rare, progressive and fatal neurodegenerative disease.
The FDA issued a complete response letter to Pfizer pertaining to the company's new drug application for tafamidis meglumine, requesting the completion of a second efficacy study to establish substantial evidence of effectiveness prior to an approval. The agency also has asked for additional information on the data within the current tafamidis NDA.
Tafamidis meglumine is a novel, investigational medication designed to treat transthyretin familial amyloid polyneuropathy, or TTR-FAP, in adult patients with symptomatic polyneuropathy to delay neurologic impairment. Currently, there is no FDA-approved treatment for this disease.
Pfizer said it will work with the FDA to address the content of the CRL.