Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the initiation of a global pivotal Phase 3 development program for fixed-dose combinations of VX-809 (lumacaftor) and ivacaftor in people with cystic fibrosis (CF) who have two copies (homozygous) of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (
) gene. Vertex plans to conduct two 24-week Phase 3 studies to support approval of the combination of VX-809 and ivacaftor in people with CF ages 12 and older. The studies, TRAFFIC and TRANSPORT, will each include two treatment groups that will evaluate VX-809 (600mg QD or 400mg q12h) in combination with ivacaftor (250mg q12h) compared to a placebo group. Vertex expects to obtain 24-week safety and efficacy data from both studies and to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) and a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA), pending study results.
Vertex also plans to conduct a pharmacokinetics and safety study to evaluate VX-809 in combination with ivacaftor in children with CF ages 6 to 11 who have two copies of the F508del mutation. The company expects to use the data from this study for subsequent registration in children ages 6 to 11 in the United States and is continuing discussions with European regulatory agencies for patients in this age group.
Vertex will host a conference call for investors and media today, February 26, 2013 at 5:15 p.m. EST, to discuss the company’s Phase 3 development plan.
“This Phase 3 development program is a significant advance in our efforts to develop new medicines that treat the underlying cause of cystic fibrosis for people with the most common type of the disease,” said Robert Kauffman, M.D., Ph.D., Senior Vice President and Chief Medical Officer at Vertex. “Importantly, these studies will evaluate two doses of VX-809 in combination with ivacaftor for 24 weeks, and pending data, enable submissions to U.S. and European regulatory authorities. People with CF are in urgent need of new treatments, and we are committed to advancing this combination through Phase 3 development as quickly as possible.”