Press release
Vertex Receives CHMP Positive Opinion for KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in Combination With Ivacaftor for Children With Cystic Fibrosis Ages 2 Through 5
-If approved, more than 1,200 children would be newly eligible for a medicine that could treat the underlying cause of their disease- BOSTON--(BUSINESS
About this update from Vertex Pharmaceuticals Incorporated
[{"type":"text","content":"\n-If approved, more than 1,200 children would be newly eligible for a medicine that could treat the underlying cause of their disease-\n\n\n BOSTON--(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 children with cystic fibrosis (CF) ages 2 through 5 years old who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.\n\n\n“KAFTRIO has demonstrated unprecedented clinical benefit for eligible people living with CF,” said Nia Tatsis, Ph.D., Executive Vice President, Chief Regulatory and Quality Officer at Vertex. “Treating the underlying cause of CF as early as possible in life has the potential to slow disease progression, which is why we are pleased the CHMP is supportive of expanding the indication for KAFTRIO to patients as young as 2 years.”\n\n\nIn the European Union, KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor is already approved for the treatment of people with CF ages 6 years and older who have at least one copy of the F508del mutation in the CFTR gene.\n\n\nAbout Cystic Fibrosis\n\n\nCystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 88,000 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 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 F508del mutation. CFTR mutations lead to CF by causing CFTR protein to be defective or by leading to a shortage or absence of CFTR protein 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 a...