Pfizer's Vyndaqel, first drug to treat rare and fatal disease TTR-FAP, approved in Europe

Following a positive opinion from an advisory committee in the summer (The Pharma Letter July 23), the European Commission has now granted final approval for US drug behemoth Pfizer’s (NYSE: PFE) Vyndaqel (tafamidis) for the treatment of the rare and fatal neurodegenerative hereditary disease transthyretin familial amyloid polyneuropathy (TTR-FAP) in adult patients with stage 1 symptomatic polyneuropathy.

“A diagnosis of TTR-FAP, usually made in patients during their mid-life years, impacts both the physical and emotional well-being of patients and caregivers and significantly limits daily activities,” said Teresa Coelho, Hospital Santo Antonio in Porto, Portugal, who participated in the clinical trials of Vyndaqel, adding: “Until now, there were no approved medications to treat this degenerative and fatal disease. Vyndaqel offers new hope to patients who are diagnosed with TTR-FAP.”

The European backing of the drug is good news for Pfizer, which received a Refusal to File letter from the US Food and Drug Administration for tafamidis, acquired with its buy last year of FoldRx (TPL April 5).