The European Commission has granted Vertex Pharmaceuticals an extension of the marketing authorization for Orkambi (lumacaftor/ivacaftor) to include children 6-11 years old who have cystic fibrosis (CF) caused by two copies of the F508del mutation in the CFTR gene.
In Europe, it is estimated that 3,400 children of that age with CF now will become eligible to receive this therapy. Orkambi was the first medicine in the European Union approved to treat the underlying cause of CF in people with genetic mutations in the CFTR gene.
CF develops due to a defective or missing CFTR protein, resulting from one of 2,000 possible mutations in the CFTR gene. People who have two copies of the F508del mutation can’t process CFTR protein normally within the cell, resulting in little-to-no CFTR protein at the cell surface.
Orkambi is a combination of lumacaftor and ivacaftor. Lumacaftor is designed to increase the amount of mature CFTR protein at the cell surface by targeting the abnormalities caused by the F508del mutation. Ivacaftor is designed to enhance the function of the CFTR protein once it reaches the cell surface.
“A principal goal of treating CF is slowing the progressive lung damage caused by this life-shortening genetic disease while improving health in the short term,” Marcus Mall, MD, director of the division of pediatric pulmonology and allergology at Heidelberg University Hospital, Germany, said in a press release.
“Studies of Orkambi in children ages 6 through 11 have shown improvements in clinically relevant outcomes, like lung function and weight gain.” Mall added.
After the approval of the marketing authorization’s extension, existing reimbursement agreements in countries like Ireland will enable rapid access to Orkambi. In the remaining EU countries, Vertex now will begin a country-by-country reimbursement process.
“The innovative long-term agreements we have reached in countries like Ireland will enable eligible children to have rapid access to Orkambi,” said Simon Bedson, senior vice president at Vertex. “Where these agreements are not in place, Vertex is committed to working with local authorities so those who could benefit from this medicine are able to do so as quickly as possible.”