Pfizer Inc. (PFE) said Monday the U.S. Food and Drug Administration has rejected its New Drug Application for tafamidis meglumine - an investigational medication for the treatment of Transthyretin Familial Amyloid Polyneuropathy in adult patients with symptomatic polyneuropathy to delay neurologic impairment.
Pfizer said the FDA has requested the completion of a second efficacy study to establish substantial evidence of effectiveness prior to an approval. The Agency also asked for additional information on the data within the current tafamidis NDA.
Transthyretin Familial Amyloid Polyneuropathy, or TTR-FAP, is a rare, progressive and fatal neurodegenerative disease, and is caused by a genetic mutation of the transthyretin gene. The disease affects about 8,000 patients worldwide. In the U.S., a non-endemic region, the incidence is estimated to be about 1 in 100,000, impacting about 3,000 people. In the U.S., there is no FDA-approved treatment for TTR-FAP. In the U.S., there is no FDA-approved treatment for TTR-FAP.
Tafamidis is a novel, selective stabilizer of the transthyretin protein approved last November by the European Commission (the trade name in the European Union and proposed trade name in the U.S. is VYNDAQEL). It is indicated in the European Union for the treatment of TTR amyloidosis in adult patients with stage 1 symptomatic polyneuropathy to delay peripheral neurologic impairment.
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