Vertex Pharma files NDA at FDA for elexacaftor, tezacaftor and ivacaftor combination for F508del mutation cystic fibrosis.

Vertex Pharmaceuticals Incorporated announced the submission of a New Drug Application (NDA) to the FDA for the VX-445 (elexacaftor), tezacaftor and ivacaftor triple combination regimen. The NDA includes a request for Priority Review, which, if granted, would shorten the FDA�s review of the NDA to eight months from the time of submission, versus a standard review timeline of 12 months from submission. The submission is supported by previously disclosed positive results of two global Phase III studies: 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.