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Applications for Tezacaftor/Ivacaftor Combination Treatment Have Been Accepted by the FDA & EMA
August 24, 2017
Today we announced the acceptance of our applications for the use of the tezacaftor/ivacaftor combination treatment in people with cystic fibrosis (CF) ages 12 and older who have two copies of the F508del mutation or one F508del mutation and one residual function mutation that is responsive to tezacaftor/ivacaftor by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). In the United States, the FDA has granted Priority Review of the New Drug Application (NDA) and set an action date of February 28, 2018. The submissions are supported by positive results from two global Phase 3 studies.
“If approved, the tezacaftor/ivacaftor combination treatment would become Vertex’s third medicine to treat the underlying cause of cystic fibrosis, offering an important new treatment option for a large group of patients with this rare and life-shortening disease,” said Jeffrey Chodakewitz, M.D., Executive Vice President and Chief Medical Officer at Vertex. “We look forward to working with the agencies to facilitate a rapid review of these applications.”