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Vertex Announces European Commission Approval for SYMKEVI® (tezacaftor/ivacaftor) With KALYDECO® (ivacaftor) for Eligible Children With Cystic Fibrosis Ages 6-11 Years
- The combination therapy is a new treatment option for CF patients who are homozygous for F508del - - The only medicine to treat the underlying cause of CF
About this update from Vertex Pharmaceuticals Incorporated
[{"type":"text","content":"\n- The combination therapy is a new treatment option for CF patients who are homozygous for F508del -\n\n- The only medicine to treat the underlying cause of CF in this age group with one F508del mutation and one of 14 residual function mutations - \n\n LONDON--(BUSINESS WIRE)--\nVertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the European Commission has granted approval of the label extension for SYMKEVI® (tezacaftor/ivacaftor) with KALYDECO® (ivacaftor), to include the treatment of cystic fibrosis (CF) in patients ages 6 years and older who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or one copy of the F508del mutation and one copy of one of 14 mutations in the CFTR gene that result in residual CFTR activity: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272-26A→G, and 3849+10kbC→T.\n\n“With this approval, children with CF in Europe ages 6 to 11 years with the most common mutation, F508del, have a new treatment option and children with certain residual function mutations will, for the first time, have a treatment option available that addresses the underlying cause of their CF,” said Reshma Kewalramani, M.D., Chief Executive Officer and President at Vertex. “Today’s approval brings us closer to our ultimate goal of providing medicines for all people with CF.”\n\nSYMKEVI® (tezacaftor/ivacaftor) with KALYDECO® (ivacaftor) will be immediately available to additional eligible patients in Germany and will be available shortly in countries that have entered into innovative long-term reimbursement agreements with Vertex, including the UK, Denmark and the Republic of Ireland. In all other countries, Vertex will work closely with relevant authorities in Europe to secure access for eligible patients.\n\nIn Europe, SYMKEVI® (tezacaftor/ivacaftor) with KALYDECO® (ivacaftor) is already approved for the treatment of people with CF ages 12 years and older who have either two copies of the F508del mutation in the CFTR gene, or one copy of the F508del mutation and a copy of one of the following 14 mutations in which the CFTR gene results in residual CFTR protein activity: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272-26A→G, and 3849+10kbC→T. \n\nAbout Cysti...