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news.Pfizer has received approval from the European Union (EU) for its Vyndaqel (tafamidis) used to treat Transthyretin Familial Amyloid Polyneuropathy (TTR-FAP) in adult patients with stage 1 symptomatic polyneuropathy.
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Vyndaqel is a transthyretin stabilizer which prevents the formation of misfolded proteins and the subsequent amyloid deposits that lead to neurodegeneration and decline of neurologic function.
A pivotal clinical trial (Fx-005) and an open-label, 12-month extension study (Fx-006), which assessed the long-term safety and efficacy of Vyndaqel in patients with TTR-FAP, led to the approval.
The data demonstrated that Vyndaqel showed efficacy in delaying peripheral neurologic impairment and improved nutritional status.