The United States Food and Drug Administration’s (FDA) Peripheral and Central Nervous System Drugs Advisory Committee has found Pfizer’s clinical data package for tafamidis meglumine. Tafamidis is a novel, investigational, oral therapy for the treatment of Transthyretin Familial Amyloid Polyneuropathy in adult patients with symptomatic polyneuropathy to delay neurologic impairment.

The Advisory Committee did not find substantial evidence of efficacy on a clinical endpoint. The Committee then voted 13-4 that the data provide substantial evidence of efficacy for a surrogate endpoint that is reasonably likely to predict a clinical benefit. This recommendation will be taken into consideration by the FDA when making its decision on Pfizer’s New Drug Application (NDA) for tafamidis as a treatment for Transthyretin Familial Amyloid Polyneuropathy (TTR-FAP), a rare and fatal neurodegenerative disease.
“TTR-FAP is an irreversible and devastating disease with no FDA-approved treatment in the US,” said Dr Yvonne Greenstreet, senior vice president and head of Medicines Development Group for Pfizer’s Specialty Care Business Unit. “The panel’s assessment represents a positive step forward in our goal to provide this much-needed medicine to patients suffering from this rare and fatal disease. Pfizer will continue to work with the FDA as the Agency finalizes its review of our NDA for tafamidis.”

TTR-FAP is a rare, progressive and fatal neurodegenerative disease that affects approximately 8,000 patients worldwide. Because it is a hereditary disease, family members may also be at risk for developing the disease. There is currently no FDA-approved treatment in the US designed specifically to treat TTR-FAP. If approved by the FDA, tafamidis would be the first and only medication in the US indicated to treat patients with this debilitating genetic disease.

The FDA has granted the tafamidis NDA both an orphan drug and a priority review designation. The Orphan Drug Designation programme provides orphan status to drugs and biologics that are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases and disorders that affect fewer than 200,000 people in the US. 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.

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. 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.

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