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EU Approves Pfizer’s Vyndaqel for Rare Neurodegenerative Disease

Stabilization was observed in 98% of patients

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By: Tim Wright

Editor-in-Chief, Contract Pharma

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 neurod...

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