Panel: Tafamidis data predictive of benefit, but efficacy evidence not robust

A panel of advisers to the US FDA on 24 May did not find substantial evidence of efficacy on a clinical endpoint in Pfizer's single study of its experimental drug tafamidis meglumine in treating transthyretin familial amyloid polyneuropathy (TTR-FAP), but said the data were strong enough to match what might be found by a surrogate endpoint likely to predict a clinical benefit.

The FDA's Peripheral and Central Nervous System Drugs Advisory Committee voted 13-4 that the findings of Pfizer's study 005 were not sufficiently robust to provide substantial evidence of efficacy similar...