Vertex Initiates Second Phase 3 VX-659 Triple Combination Study in Certain People with Cystic Fibrosis
Vertex Initiates Phase 3 Study of VX-659, Tezacaftor and Ivacaftor as a Triple Combination Regimen for People with Cystic Fibrosis Who Have Two Copies of the F508del Mutation
March 1, 2018
We announced the initiation of a Phase 3 study of VX-659, tezacaftor and ivacaftor as an investigational triple combination regimen for people with cystic fibrosis (CF) who have two copies of the F508del mutation, the most common genetic form of the disease. The study will enroll approximately 100 patients, and the primary endpoint of the study is the mean absolute change from baseline in percent predicted forced expiratory volume in one second (ppFEV1) at week four of treatment. The study is designed to support the submission of an application for approval in patients with two copies of the F508del mutation in the U.S. using data from the 4-week primary efficacy endpoint together with 24-week safety data generated from the recently initiated Phase 3 study in patients with one F508del mutation and one minimal function mutation. Learn more about this investigational program here.