Therapy Areas: Hereditary Disorders
European Commission grants approval to Vertex Pharmaceuticals (Europe) for Orkambi
23 January 2019 -

The European Commission (EC) has granted approval to the United Kingdom-based Vertex Pharmaceuticals (Europe) for Orkambi (lumacaftor/ivacaftor) intended for the treatment of children with cystic fibrosis (CF) aged two to five years old who have two copies of the F508del mutation, it was reported yesterday.

Lumacaftor/ivacaftor has already secured approval in the EU to treat CF in patients aged six and older who have two copies of the F508del mutation. The label extension has been granted to treat the most common form of CF, based on data from a phase three open-label safety study in 60 patients. The company said that the data demonstrated that treatment with Orkambi 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.

The product is a combination of lumacaftor, which is developed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del-CFTR protein, while ivacaftor has been developed to improve the function of the CFTR protein once it reaches the cell surface.