FDA review says tafamidis lacks evidence of benefit.

The Wall Street Journal (5/23, Dooren, Subscription Publication) reports that a Food and Drug Administration review in advance of Thursday's meeting of the Peripheral and Central Nervous Systems Drugs Advisory Committee finds a lack of evidence that tafamidis (Pfizer, Vyndaqel) benefits patients with transthyretin familial amyloid polyneuropathy, an inherited and fatal condition. The medication was approved last year for sale in Europe. The review cited a study in which the medication failed to meet its primary goal, but did meet at least one secondary goal.

        Reuters (5/23, Yukhananov) reports that most patients die within 11 years of the onset of symptoms and that a liver transplant is the only current treatment.

        MedPage Today (5/23, Walker) explains, "Familial amyloid polyneuropathy (FAP) is caused by mutations of the transthyretin (TTR) gene, which leads to build up of amyloid primarily in the peripheral nerves, as well as other organs. Symptoms usually start between the age of 30 and 50 and include sensory loss that can be accompanied with severe pain as well as severe autonomic dysfunction." It "tends to be clustered in Portugal, Sweden, and Japan," though "there are estimated to be about 2,500 patients with familial amyloid polyneuropathy" in the US.