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Vertex Receives CHMP Positive Opinion for Expanded Label for KAFTRIO® in Combination With Ivacaftor for People With Cystic Fibrosis to Include Rare Mutations
-If approved, approximately 4,000 people with cystic fibrosis in the European Union will be eligi...
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[{"type":"text","content":"Vertex Receives CHMP Positive Opinion for Expanded Label for KAFTRIO® in Combination With Ivacaftor for People With Cystic Fibrosis to Include Rare Mutations\n\n\n .bwalignc { text-align: center; list-style-position: inside }\n \n\n\n\n\n -If approved, approximately 4,000 people with cystic fibrosis in the European Union will be eligible for a medicine that treats the underlying cause of their disease for the first time-\n \n\n\n\n\n\n Vertex Pharmaceuticals\n \n (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 expansion of KAFTRIO\n \n ®\n \n (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor for the treatment of people with cystic fibrosis (CF) aged 2 years and older who have at least one non-class I mutation in the cystic fibrosis transmembrane conductance regulator (\n \n CFTR\n \n ) gene.\n \n\n “We are thrilled by the positive CHMP opinion in support of expanding the KAFTRIO in combination with ivacaftor indication to include all CF patients 2 years and older who make CFTR protein,” said Fosca De Iorio, M.D., Vice President, International Medical Affairs at Vertex. “If approved, thousands of additional patients across Europe will be eligible for a CFTR modulator, bringing us closer than ever to our goal of getting treatments that address the underlying cause of the disease to all people living with CF.”\n \n\n In the European Union, ivacaftor/tezacaftor/elexacaftor in combination with ivacaftor is currently approved for the treatment of people with CF aged 2 years and older who have at least one copy of the\n \n F508del\n \n mutation in the\n \n CFTR\n \n gene.\n \n\n\n About Cystic Fibrosis\n \n\n\n Cystic 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\n \n CFTR\n \n gene. Children must inherit two defective\n \n CFTR\n \n genes — one from each parent — to have CF, and these mutations can be identified by a genetic test....