Vertex Pharmaceuticals wants to submit a New Drug Application in the USA, so it will soon give the final test VX-659, tezacaftor and ivacaftor as an investigational triple combination regimen for people with cystic fibrosis (CF) who have one F508del mutation and one minimal function mutation.
The company said that the Phase 3 study kicks off with 360 patients to reach the mean absolute change from baseline in percent predicted forced expiratory volume in one second (ppFEV1) at week four of treatment.
“Our goal is to bring the best triple combination to patients as rapidly as possible, and this first Phase 3 study of VX-659 in combination with tezacaftor and ivacaftor is a significant step toward that goal,” said Jeffrey Chodakewitz, Executive Vice President and Chief Medical Officer at Vertex.
Steven M. Rowe, the Director of the Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama said there is a significant unmet medical need to treat the underlying cause of CF for those with one F508del mutation and a minimal function mutation. “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,” said Rowe, who is co-chair of Vertex’s Triple Combination Steering Committee-