FDA committee backs Vertex's Orkambi
FDA's Pulmonary-Allergy Drugs Advisory Committee voted 12-1 on Tuesday that available efficacy and safety data support approval of Orkambi, a combination of lumacaftor ( VX-809) and Kalydeco ivacaftor from Vertex Pharmaceuticals Inc. (NASDAQ:VRTX), to treat cystic fibrosis patients homozygous for the F508 CFTR mutation. The panel's consumer representative cast the lone dissenting vote. Orkambi has breakthrough therapy designation from FDA and is under Priority Review, with a July 5 PDUFA date. The F508 CFTR mutation is the most common mutation among CF patients.
Last June, Vertex said Orkambi met the primary endpoint of improving the absolute change from baseline in percent predicted forced expiratory volume in one second (ppFEV1) at week 24 vs. placebo in the identical Phase III TRAFFIC and TRANSPORT trials (see BioCentury, June 30, 2014). ...