NEW YORK CITY (dpa-AFX) - Pfizer, Inc. (PFE) announced that the European Commission has approved Vyndaqel (tafamidis) for treating Transthyretin Familial Amyloid Polyneuropathy, or TTR-FAP, in adult patients with stage 1 symptomatic polyneuropathy. TTR-FAP is a rare, progressive and fatal neurodegenerative disease.
Pfizer noted that the approval is based on results from 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. Across the above clinical studies, Vyndaqel showed efficacy in delaying peripheral neurologic impairment. Extra data from the studies showed 51 to 81 percent less deterioration in neurologic function, large fiber function and small fiber function versus patients treated with placebo.
It was further observed in the studies that Vyndaqel resulted in improved nutritional status. Also, decline in mBMI was shown to correlate with disease progression in the pivotal 18-month study.
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