Vertex gains 55% on surprising interim data from Kalydeco combo in cystic fibrosis
This article was originally published in Scrip
Executive Summary
Vertex Pharmaceuticals, which has already succeeded in serving a niche population in cystic fibrosis with its recently approved drug Kalydeco (ivacaftor), has now opened the way to a much broader market in cystic fibrosis by pairing Kalydeco with the experimental agent VX-809. Interim results of a Phase II trial showed that the combination significantly improved lung function in adults who have two copies (homozygous) of the F508del cystic fibrosis transmembrane conductance regulator (CFTR) gene.