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Vertex Announces European Commission Approval for KALYDECO® (ivacaftor) as First and Only CFTR Modulator to Treat Eligible Infants With Cystic Fibrosis as Early as Four Months of Age
- Approval provides opportunity to treat the underlying cause of cystic fibrosis earlier than ever before in Europe - LONDON--(BUSINESS WIRE)-- Vertex
About this update from Vertex Pharmaceuticals Incorporated
[{"type":"text","content":"\n- Approval provides opportunity to treat the underlying cause of cystic fibrosis earlier than ever before in Europe -\n\n LONDON--(BUSINESS WIRE)--\nVertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the European Commission has granted approval of the label extension for KALYDECO® (ivacaftor) granules to include the treatment of infants with cystic fibrosis (CF) ages 4 months and older and weighing at least 5 kg who have the R117H mutation or one of the following gating (class III) mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R.\n\n“Our very first CFTR modulator, KALYDECO, was first approved eight years ago, for certain CF patients ages 6 years and older. With today’s approval, babies as young as 4 months are eligible and we believe early treatment is important in managing CF,” said Reshma Kewalramani, M.D., Chief Executive Officer and President, Vertex. “Today’s approval is a testament to our commitment to keep going until all people with CF have a treatment option.”\n\nThe label update is based on data from a cohort in the 24-week Phase 3 open-label safety study (ARRIVAL) consisting of six children with CF ages four months to less than six months who have eligible gating mutations.\n\nKALYDECO® (ivacaftor) will be now 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\nKALYDECO® (ivacaftor) is already approved in Europe for people with CF ages 6 months and older weighing at least 5 kg who have one of the following mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, R117H, S1251N, S1255P, S549N or S549R.\n\nAbout Cystic Fibrosis \n\nCystic 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. Chi...