Vertex Pharmaceuticals (Europe) Limited has announced that the European Medicines Agency (EMA) has validated a Marketing Authorization Application (MAA) for the VX-445 (elexacaftor), tezacaftor and ivacaftor triple combination regimen for cystic fibrosis.
The submission was supported by previously disclosed positive results of two global Phase 3 studies in people with cystic fibrosis (CF): a 24-week study in people with one F508del mutation and one minimal function mutation and a 4-week study in people with two F508del mutations.
Both Phase 3 studies showed statistically significant improvements in lung function (percent predicted forced expiratory volume in one second; ppFEV1), which was the primary endpoint, and in all key secondary endpoints. In these studies, the triple combination regimen was generally well tolerated.
“Today marks a significant milestone towards our efforts to bring new medicines to more people around the world who are living with cystic fibrosis,” said Reshma Kewalramani, M.D., Executive Vice President and Chief Medical Officer at Vertex. “We are looking forward to working with the EMA on this important application.”