Pfizer received approval for Vyndaqel (tafamidis) from the European Commission for the treatment of Transthyretin Familial Amyloid Polyneuropathy (TTR-FAP) in adult patients with stage 1 symptomatic polyneuropathy. TTR-FAP is a rare, progressive and fatal neurodegenerative disease that affects approximately 8,000 patients worldwide.

Vyndaqel is a novel specific transthyretin stabilizer designed to prevent the formation of misfolded proteins and the subsequent amyloid deposits that induce neurodegeneration and decline of neurologic function. In the trial (Fx-005), transthyretin stabilization was observed in 98% of patients on Vyndaqel, and in no patients on placebo, at 18 months.

“Today marks a real breakthrough for patients in the EU living with TTR-FAP,” said Yvonne Greenstreet, senior vice president and head of Medicines Development Group for Pfizer’s Specialty Care Business Unit. “This community urgently needs an effective therapy, and we are proud to be able to provide the first and only approved medication for patients with this rare and debilitating genetic disease. Pfizer is focused on meeting the needs of patients suffering from rare diseases and this approval is an important step forward in our commitment to providing treatment options for patients.”