Vertex Pharmaceuticals Incorporated announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (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 3 studies: 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.
“We have relentlessly focused on the progression of VX‑445 (elexacaftor), tezacaftor and ivacaftor from discovery through clinical development and regulatory submission,” said Reshma Kewalramani, M.D., Executive Vice President and Chief Medical Officer at Vertex. “The submission of the NDA is a major step toward our goal of bringing this medicine to the largest remaining group of people with CF that still do not have an approved Vertex medicine, as well as toward providing significantly enhanced benefits to patients with two F508del mutations. We will continue working with the FDA as they review the NDA and look forward to the potential of this triple combination regimen becoming a new treatment option for people with CF.”
In the United States, the VX-445 (elexacaftor), tezacaftor and ivacaftor treatment regimen was granted Breakthrough Therapy designation in May 2018. Breakthrough Therapy designation is a process designed to expedite the development and review of drugs that are intended to treat a serious condition and for which preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s).