FDA Issues Complete Response Letter to NDA for Tafamidis Meglumine

Pfizer announced that it has received a Complete Response Letter from the FDA on its New Drug Application (NDA) for tafamidis meglumine for the treatment of Transthyretin Familial Amyloid Polyneuropathy (TTR-FAP) in adult patients with symptomatic polyneuropathy to delay neurologic impairment.

The letter states that FDA is requesting the completion of a second efficacy study to establish substantial evidence of effectiveness prior to an approval. The Agency has also asked for additional information on the data within the current tafamidis NDA. Pfizer will work with the FDA to address the content of the letter.

TTR-FAP is a rare, progressive, and fatal neurodegenerative disease that affects approximately 8,000 patients worldwide. TTR-FAP is primarily caused by a genetic mutation of the transthyretin (TTR) gene. 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.

Tafamidis is a novel, selective stabilizer of the transthyretin protein.

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