Tafamidis: The First Drug Approved for a Rare Neurodegenerative Disease

Transthyretin Familial Amyloid Polyneuropathy (TTR-FAP) is a rare, progressive and fatal neurodegenerative disease that affects approximately 8,000 patients worldwide. Based on results from a pivotal 18-month placebo controlled clinical trial featuring an 18-month follow-on study, Pfizer’s tafamidis (Vyndaqel) has proven its effectiveness in delaying peripheral neurologic impairment. In giving the drug its green light, the European Commission made tafamidis the world’s first approved medication for TTR-FAP. More information and online registration is available here.