A rare win

Pfizer Inc. saw value in a failed Phase II/III trial of tafamidis as a treatment for transthyretin familial amyloid polyneuropathy when it acquired FoldRX Pharmaceuticals Inc. in 2010. Last week, FDA's Peripheral and Central Nervous System Drugs Advisory Committee agreed, voting 13-4 that data on surrogate endpoints in the study were sufficiently robust to predict clinical benefit.

The vote paves the way for accelerated approval in the Orphan disease...