Press release
Vertex Receives CHMP Positive Opinion for KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in Combination With Ivacaftor in Children With Cystic Fibrosis Ages 6 Through 11
- If approved, more than 1,500 children would be eligible for a medicine that can treat the underlying cause of their disease for the first time -
About this update from Vertex Pharmaceuticals Incorporated
[{"type":"text","content":"\n- If approved, more than 1,500 children would be eligible for a medicine that can treat the underlying cause of their disease for the first time -\n\n LONDON--(BUSINESS WIRE)--\nVertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for the label extension of KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor, for the treatment of cystic fibrosis (CF) in patients ages 6 through 11 years old who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.\n\n“Today marks an important milestone for the treatment of children with CF in Europe. If approved, KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor will offer physicians a new treatment option for these young patients to help combat this life-shortening condition as early as possible,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex.\n\n“The clinical data for ivacaftor/tezacaftor/elexacaftor plus ivacaftor in people with CF ages 6 through 11 with eligible CF genotypes demonstrated improvements in lung function, sweat chloride and respiratory symptoms and a safety and tolerability profile consistent with that observed in patients ages 12 years and older,” said Professor Marcus A. Mall, M.D., Head of the Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine at Charité University Medical Center Berlin. “This medicine has already made a big impact on the lives of eligible people ages 12 years and above in Europe. The CF community is now looking forward to it being available for younger patients too, to enable treatment as early as possible in life.”\n\nAbout Cystic Fibrosis\n\nCystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 83,000 people globally. 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 m...