EMA validates MAA for VX-445 triple combination treatment in cystic fibrosis -Vertex

Vertex Pharmaceuticals has announced that the European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for the VX 445 (elexacaftor), tezacaftor and ivacaftor triple combination regimen. The submission was supported by previously disclosed positive results of two global Phase III 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 III 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.