European Commission approves Orkambi for the treatment of children with cystic fibrosis aged 2 to 5 years old who have two copies of the F508del mutation. - Vertex Pharma

Vertex Pharmaceuticals (Europe) Limited announced that the European Commission has granted approval of the label extension for Orkambi (lumacaftor/ivacaftor) for the treatment of children with cystic fibrosis (CF) aged 2 to 5 years old who have two copies of the F508del mutation, the most common form of the disease. The label update is based on data from a Phase III open-label safety study in 60 patients that showed treatment with lumacaftor/ivacaftor was generally well tolerated for 24 weeks, with a safety profile in these pediatric patients generally consistent with that in patients aged 6 years and older. Lumacaftor/ivacaftor is already approved in the EU for the treatment of CF in patients aged 6 and older who have two copies of the F508del mutation.