Press release
European Commission Approves Expanded Label for KAFTRIO® in Combination With Ivacaftor for People With Cystic Fibrosis
-Approximately 4,000 people living with CF in the European Union are newly eligible for a medicine that treats the underlying cause of their disease for the
About this update from Vertex Pharmaceuticals Incorporated
[{"type":"text","content":"\n-Approximately 4,000 people living with CF in the European Union are newly eligible for a medicine that treats the underlying cause of their disease for the first time-\n\n LONDON--(BUSINESS WIRE)--\nVertex Pharmaceuticals (Nasdaq: VRTX) today announced that the European Commission has granted regulatory approval for a label expansion of KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor for the treatment of people with cystic fibrosis (CF). With this approval, the indication has been expanded to include all patients ages 2 years and older who have at least one non-class I mutation in the cystic fibrosis conductance regulator (CFTR) gene.\n\n“We have been working for years to bring treatment options to all people with cystic fibrosis, including those with ultra rare mutations,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer, Vertex. “We are pleased that the European Commission has broadened the KAFTRIO indication to include all CF patients 2 years and older who have at least one non-class I mutation, ensuring that even more people living with CF can benefit from this transformative medicine.”\n\nAs a result of existing KAFTRIO reimbursement agreements in Austria, Denmark, Ireland, Norway and Sweden, and provisions for access in health care systems such as Germany, eligible patients in these countries will have access to the expanded indication of the therapy shortly.\n\nVertex will continue to work collaboratively with reimbursement authorities across the European Union to ensure access for all eligible patients, as quickly as possible.\n\nAbout Cystic Fibrosis\n\nCystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 94,000 people in North America, Europe and Australia. CF is a progressive, multi-organ disease that affects the lungs, liver, pancreas, GI tract, sinuses, sweat glands and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF, and these mutations can be identified by a genetic test. While there are many different types of CFTR mutations that can cause the disease, the vast majority of people with CF have at least one F50...