Pfizer Inc. announced that the FDA has accepted its New Drug Application (NDA) for tafamidis meglumine for the treatment of Transthyretin Familial Amyloid Polyneuropathy (TTR-FAP). The FDA has granted the tafamidis NDA a priority-review designation.
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 TTR protein. 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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