Vertex Pharmaceuticals Inc (VRTX)

[surf1944]

4:22PM Vertex Pharm announces initiation of pivotal Phase 3 program of VX-809 in combination with ivacaftor for the treatment of people with cystic fibrosis who have two copies of the F508del mutation (VRTX) 44.18 -0.68 : Co 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 (CFTR) 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 to the FDA and a Marketing Authorization Application (MAA) to the European Medicines Agency, pending study results.

Co 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.