The US Food and Drug Administration (FDA) has accepted for review of Pfizer Inc.'s New Drug Application (NDA) for tafamidis meglumine, the investigational, novel, oral therapy for the treatment of Transthyretin Familial Amyloid Polyneuropathy (TTR-FAP).

TTR-FAP is a rare, progressive and fatal neurodegenerative disease that affects approximately 8,000 patients worldwide. The European Commission approved tafamidis (the trade name in the European Union is Vyndaqel) in November 2011.

The US FDA has granted the tafamidis NDA a priority-review designation and has provided an anticipated Prescription Drug User Fee Act (PDUFA) action date in June 2012. Priority-review designation is granted to drugs that have the potential to offer significant improvement in treatment or provide a treatment where no adequate therapy exists.

Transthyretin Familial Amyloid Polyneuropathy (TTR-FAP) is a rare and fatal neurodegenerative disease 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.3,4 Patients with TTR-FAP experience significantly diminished quality of life due to symptoms including polyneuropathy characterized by sensory loss, pain and weakness in the lower limbs; as well as severe impairment of the autonomic nervous system commonly manifesting as erectile dysfunction, alternating diarrhoea and constipation, unintentional weight loss, orthostatic hypotension, urinary incontinence, urinary retention and delayed gastric emptying. As the disease progresses, patients often lose the ability to walk, needing wheelchair assistance, and eventually become bedridden and unable to care for themselves. TTR-FAP typically occurs during active adult years with onset as early as the 30s, followed by disease progression that may reach the terminal stage in approximately 10 years on average.

Tafamidis is a novel, selective stabilizer of the TTR protein approved in November 2011 by the European Commission. 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. The drug is currently under review by the FDA in the US.

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