Posted by Michael Wonder on 22 Jan 2019
Vertex announces European Commission approval for Orkambi (lumacaftor/ivacaftor) for treatment of children with cystic fibrosis aged 2 to 5 years old with most common form of the disease
21 January 2019 - Lumacaftor/ivacaftor is the first and only approved medicine in Europe to treat the underlying cause of cystic fibrosis for approximately 1,500 children aged 2 to 5 with two copies of the F508del mutation.
Vertex Pharmaceuticals today announced that the European Commission has granted approval of the label extension for Orkambi (lumacaftor/ivacaftor) for the treatment of children with cystic fibrosis 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 3 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 paediatric patients generally consistent with that in patients aged 6 years and older.
