Spanish Government Approves National Reimbursement of ORKAMBI (lumacaftor/ivacaftor) and SYMKEVI (tezacaftor/ivacaftor) in Combination With KALYDECO (ivacaftor)
Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) today announced that the Spanish Government has approved terms for the national reimbursement of ORKAMBI (lumacaftor/ivacaftor) and SYMKEVI (tezacaftor/ivacaftor) in combination with KALYDECO (ivacaftor) for eligible patients in Spain living with cystic fibrosis (CF).
Under the terms announced today, children ages 6 to 11 years with CF who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene can be treated with ORKAMBI. Patients ages 12 years and older who either have two copies of the F508del mutation, or one copy of the F508del mutation and a copy of one of the other 14 mutations approved within the license in which the CFTR protein shows residual activity, can be treated with SYMKEVI in combination with KALYDECO.
Ludovic Fenaux, Senior Vice President, Vertex International, commented, “Today’s announcement means ORKAMBI and SYMKEVI can be prescribed from the first of November 2019 for the approximately 600 eligible cystic fibrosis patients living in Spain, to treat the underlying cause of their disease. We thank the Spanish Authorities for their collaboration and commitment to working with us in an innovative way to come to a solution for patients. We would also like to acknowledge the medical community for their important input during this process.”
Vertex’s CF medicines are reimbursed in 17 countries around the world including Austria, Australia, Denmark, Germany, the Republic of Ireland, Italy, the Netherlands, Sweden and the U.S.
Cystic Fibrosis (CF) is a rare, life-shortening genetic disease affecting approximately 75,000 people worldwide. CF is a progressive, multi-system disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas 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. While there are many different types of CFTR mutations that can cause the disease, the vast majority of all people with CF have at least one F508del mutation. These mutations, which can be determined by a genetic test or genotyping test, lead to CF by creating non-working and/or too few CFTR proteins at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the early 30s.