Vertex Pharmaceuticals affirmed initiating the first Phase 3 study of VX-659, tezacaftor and ivacaftor as an investigational triple combination regimen for people with cystic fibrosis (CF) who have one F508delmutation and one minimal function mutation.
The study set to enlist 360 patients, and the primary endpoint of the study is the mean absolute change from baseline in percent predicted forced expiratory volume in one second at week four of treatment. The study further advocates the submission of New Drug Application in the U.S using data from the 4-week primary efficacy endpoint.
Jeffrey Chodakewitz, M.D., Executive Vice President and Chief Medical Officer at Vertex said “We’re pleased to initiate this study and look forward to working closely with the CF community to advance our two different triple combination regimens through Phase 3 development.”
Steven M. Rowe, M.D., M.S.P.H., Director of the Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham, and co-chair of Vertex’s Triple Combination Steering Committee said “The Phase 2 data for the triple combination of VX-659, tezacaftor and ivacaftor showed impressive improvements in multiple measures of CF for patients with minimal function mutations, and I am pleased that this Phase 3 study is designed to enable rapid advancement of the VX-659 regimen toward patients.”