Lumacaftor: Completed Phase III enrollment

Vertex completed enrollment of about 559 CF patients ages >=12 years who are homozygous for the delta F508 CFTR mutation in the double-blind, placebo-controlled, international Phase III TRAFFIC trial evaluating once-daily 600 mg or twice-daily 400 mg VX-809 plus twice-daily 250 mg Kalydeco ivacaftor for 24 weeks. Vertex also completed enrollment of about 563 patients in the identical Phase III TRANSPORT trial. The company plans to submit an NDA to FDA and an MAA to EMA for the combination treatment in 2H14.

The trial includes a rollover, double-blind extension study in which all patients will receive 1 of the combination regimens for up to an additional 96 weeks. Vertex also said it completed dosing in the pharmacokinetics portion of a Phase II pharmacokinetics and safety trial to evaluate VX-809 plus Kalydeco for up to 24 weeks in children aged 6-11 who are homozygous for the F508del mutation. Enrollment in the second part of the trial is expected to begin next quarter. Vertex expects to use data from the trial for subsequent registration of the combination in children ages 6-11 years in the U.S. FDA is not requiring an efficacy trial in these patients. ...