EC approves expanded label for Orkambi

The European Commission has granted approval of the label extension for Orkambi (lumacaftor/ivacaftor) for the treatment of children with cystic fibrosis (CF) aged two to five years old who have two copies of the F508del mutation, the most common form of the disease, the drug’s developer, Vertex Pharmaceuticals (Nasdaq: VRTX), announced today.

“Today’s approval by the European Commission brings us one step closer to our goal of bringing treatment to all people living with CF,” said Dr Reshma Kewalramani, executive vice president, global medicines development and medical affairs and chief medical officer at Vertex. ‘‘By treating the underlying cause of disease early, we can potentially modify its course and offer patients the chance of improved outcomes,’’ he noted.

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 six years and older.