Vyndaqel(Pfizer) is EU approved for TTR-FAP

Vyndaqel (tafamidis), an orphan medicine from Pfizer to treat transthyretin familial amyloidosis in adult patients with symptomatic polyneuropathy (TTR-FAP), a neurogenerative disease, is EU approved The approval is based on studies that demonstrated the novel specific transthyretin stabiliser showed efficacy in delaying peripheral neurologic impairment, plus 51%-81% less deterioration in neurologic function, plus large and small fibre function compared with patients treated with placebo. Vyndaqel resulted in "improved nutritional status", while the adverse drug reactions were diarrhoea, upper abdominal pain, urinary tract infection and vaginal infection. Pfizer said it is working closely with the relevant national health authorities across the EU to launch the new treatment by early 2012. The drug received a Refusal to File letter from the FDA in March this year.