Successful Phase III study of Orkambi (lumacaftor/ivacaftor) in children who carry two copies of F508del mutation cystic fibrosis.- Vertex Pharma

Vertex Pharmaceuticals Incorporated announced the results of a Phase III study of Orkambi (lumacaftor/ivacaftor) in children with cystic fibrosis (CF) 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 (LCI 2.5) through 24 weeks of treatment, demonstrating a statistically significant improvement in LCI 2.5 among patients treated with Orkambi compared to placebo.

LCI is a sensitive measure of lung function in early CF disease and the European Medicines Agency (EMA) agreed to the primary endpoint for this study. In the first half of 2017, Vertex plans to submit a Marketing Authorization Application (MAA) line extension to the EMA for the use of Orkambi in this patient population. Data from a previous Phase III open-label safety study in children ages 6 through 11 supported the FDA approval of Orkambi in September 2016 . In this second study, Orkambi was well tolerated with safety data that were similar to data from the previous Phase III study.

Comment: here are approximately 3,400 children ages 6 through 11 who have two copies of the F508del mutation in Europe.