Vertex Pharma: ORKAMBI Study Meets Endpoint In Children With Cystic Fibrosis

(RTTNews) - Vertex Pharmaceuticals Inc. (VRTX) announced the results of a Phase 3 study of ORKAMBI (lumacaftor/ivacaftor) in children with cystic fibrosis ages 6 through 11 who have two copies of the F508del mutation. The study met its primary endpoint of absolute change in lung clearance index (LCI2.5) through 24 weeks of treatment, demonstrating a statistically significant improvement in LCI2.5 among patients treated with ORKAMBI compared to placebo. In the study, ORKAMBI was well tolerated with safety data that were similar to data from the previous Phase 3 study.

Jeffrey Chodakewitz, Chief Medical Officer at Vertex, stated: "We are preparing to submit these important data to the EMA in the first half of 2017, and we look forward to bringing ORKAMBI to eligible children in Europe as soon as possible."