Vertex Pharmaceuticals Limited will extend label for its cystic fibrosis children patients treatment Orkambi in Europe, after the Union’s Commissions’ approval.
So far, Orkambi, (lumacaftor/ivacaftor) has been approved in the EU for the treatment of cystic fibrosis for children aged 6 and older, and not it gets to be available for patients aged 2 to 5 with the same form of the disease, which is the most common.
Reshma Kewalramani, Executive Vice President, Global Medicines Development and Medical Affairs and Chief Medical Officer at Vertex, said: “Today’s approval by the European Commission brings us one step closer to our goal of bringing treatment to all people living with CF. By treating the underlying cause of disease early, we can potentially modify its course and offer patients the chance of improved outcomes.’’
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 pediatric patients generally consistent with that in patients aged 6 years and older.