Posted by Michael Wonder on 20 Aug 2019
FDA accepts new drug application for VX-445 (elexacaftor), tezacaftor and ivacaftor combination treatment
20 August 2019 - FDA grants priority review of the application and sets a PDUFA target action date of 19 March 2020.
Vertex Pharmaceuticals today announced the U.S. FDA accepted its new drug application for the VX-445 (elexacaftor), tezacaftor and ivacaftor triple combination regimen.
The submission was supported by previously disclosed positive results of two global Phase 3 studies in people with cystic fibrosis (CF): a 24-week Phase 3 study in people with one F508del mutation and one minimal function mutation and a 4-week Phase 3 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.
