Vertex Pharmaceuticals (Europe) has secured approval from the European Commission (EC) for Orkambi (lumacaftor/ivacaftor) to treat of children with cystic fibrosis (CF) aged two to five years old 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 3 open-label safety study in 60 patients.
According to the company, the data demonstrated that treatment with Orkambi 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.
In addition, improvements in sweat chloride, which is a secondary endpoint, have been notieced at week 24, said the company.
Cystic fibrosis is a rare and life-shortening genetic disease, which is said to affect around 75,000 people in North America, Europe and Australia.
Lumacaftor/ivacaftor was already secured approval in the EU to treat CF in patients aged six and older who have two copies of the F508del mutation.
Orkambi 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 is developed to enhance the function of the CFTR protein once it reaches the cell surface.
Vertex chief medical officer and global medicines development and medical affairs executive vice president Dr Reshma Kewalramani 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.’’
Vertex is a global biotechnology firm engaged in the development of advanced medicines to treat serious and life-threatening diseases. The company has over dozen ongoing research programs focused on the underlying mechanisms of other serious diseases, in addition to clinical development programs in CF.
The firm manages research and development sites and commercial offices in the US, Europe, Canada, Australia and Latin America.