The European Commission has expanded use of Orkambi to include children with cystic fibrosis (CF) aged two years to five years.
The drug was initially approved in Europe in 2015 for patients with CF ages 12 years and older, and was later expanded to include children ages six to 11 years.
Orkambi (lumacaftor/ivacaftor) is the only approved medicine in Europe to treat the underlying cause of CF for the approximately 1500 children aged two to five years needing treatment. The drug is appropriate for patients who have two copies of the F508del mutation, the most common form of the disease.
The decision comes based off the back of a Phase III open-label safety study in 60 patients that showed treatment with Orkambi was generally well tolerated for 24 weeks, with a safety profile similar to that in patients aged 6 years and older.
The approval "brings us one step closer to our goal of bringing treatment to all people living with CF,” said 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.’’
Cystic fibrosis is a rare, life-shortening genetic disease affecting approximately 75,000 people in North America, Europe and Australia, caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene.