Vertex Pharmaceuticals got European Commission’s grant to extend the Marketing Authorization for ORKAMBI, the first medicine to treat the basic cause of cystic fibrosis (CF) in certain patients.
Namely, the people with two copies of the F508del mutation, to include children ages 6 through 11.
As Vertex points out, there are about 3,400 children ages 6 through 11 with two copies of this mutation in Europe. Existing reimbursement agreements in countries such as Ireland will enable rapid access to ORKAMBI for these children. In other countries across the European Union, Vertex said it will now begin the 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 and International General Manager 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.”
Professor Marcus Mall, Director of the Division of Pediatric Pulmonology & Allergology and the Cystic Fibrosis Center at the Heidelberg University Hospital, Germany, said: “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. Studies of ORKAMBI in children ages 6 through 11 have shown improvements in clinically relevant outcomes, like lung function and weight gain.”