FDA accepts elexacaftor, tezacaftor and ivacaftor, triple therapy, to treat cystic fibrosis and assigned a PDUFA date of 19 March 2020

Vertex Pharmaceuticals Incorporated announced the FDA accepted its New Drug Application (NDA) for the VX-445 (elexacaftor), tezacaftor and ivacaftor triple combination regimen. The FDA has granted Priority Review of the NDA and assigned a Prescription Drug User Fee Act (PDUFA) target action date of March 19, 2020.

The submission was supported by previously disclosed positive results of two global Phase III studies in people with cystic fibrosis (CF): a 24-week Phase III study in people with one F508del mutation and one minimal function mutation and a 4-week Phase III 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.