FDA panel to discuss Vertex CF combo

FDA's Pulmonary-Allergy Drugs Advisory Committee will meet on May 12 to discuss an NDA from Vertex Pharmaceuticals Inc. (NASDAQ:VRTX) for a combination of lumacaftor ( VX-809) and Kalydeco ivacaftor to treat cystic fibrosis (CF) in patients aged 12 and older with two copies of the F508 CF transmembrane conductance regulator ( CFTR) mutation. FDA is reviewing the NDA under Priority Review, with a July 5 PDUFA date.

Lumacaftor is a small molecule CFTR corrector, while Kalydeco is a small molecule potentiator of CFTR. The combination, which has breakthrough therapy designation from FDA, met the primary endpoint of two Phase III trials last year (see BioCentury, June 30, 2014). ...