First medicine for treatment of underlying cause cystic fibrosis approved.

Vertex Pharmaceuticals Incorporated announced that the European Commission has granted extension of the Marketing Authorization for Orkambi (lumacaftor/ivacaftor), the first medicine to treat the underlying cause of cystic fibrosis (CF) in people with two copies of the F508del mutation, to include children ages 6 through 11.

In Europe , there are approximately 3,400 children ages 6 through 11 with two copies of this mutation. Existing reimbursement agreements in countries such as Ireland will enable rapid access to Orkambi for these children. In other countries across the European Union , Vertex will now begin the country-by-country reimbursement process.

The European Commission's decision is based on data from two Phase III studies of Orkambi in children with CF ages 6 through 11 who have two copies of the F508del mutation. In 2017, The Lancet Respiratory published 24-week data from one of these studies, which demonstrated statistically significant improvements in lung function (as assessed by the absolute change in lung clearance index, or LCI2.5, and predicted forced expiratory volume in one second, or ppFEV1) among children treated with Orkambi compared to placebo. Improvements in body mass index (BMI) and the Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain score were also observed, although they were not statistically significant. Safety data were similar to those observed in an earlier Phase III open-label safety study in children ages 6 through 11.