Vertex Receives European CHMP Positive Opinion for KALYDECO® (ivacaftor) for Children and Adolescents With Cystic Fibrosis Between the Ages 6 Months and 18 Years With the R117H Mutation in the CFTR Gene

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[{"type":"text","content":"\n- If approved, KALYDECO® (ivacaftor) will be the first and only medicine in Europe to treat the underlying cause of cystic fibrosis in patients with the R117H mutation, the most common residual function mutation, as young as 6 months of age -\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 KALYDECO® (ivacaftor), to include the treatment of children and adolescents with cystic fibrosis (CF), ages 6 months and older weighing at least 5 kg who have the R117H mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.\n\n\n“Today’s announcement is important for young people with CF, as early intervention and treatment of this devastating and progressive disease is key to keeping patients healthier longer,” said Carmen Bozic, M.D., Executive Vice President and Chief Medical Officer at Vertex. “This milestone also brings us one step closer to achieving our ultimate goal of bringing medicines forward to all people with CF.”\n\n\nThe European Commission will now review the CHMP’s positive opinion, and should they issue a favorable adoption, KALYDECO® (ivacaftor) will be the first and only approved medicine in Europe to treat the underlying cause of CF in patients ages 6 months and older with the R117H mutation. In countries where long-term reimbursement agreements have been secured, KALYDECO® (ivacaftor), if approved, would be available to eligible patients shortly after Marketing Authorization. In Germany, the medicine would be available at Marketing Authorization. In all other countries, we will work closely with relevant authorities in Europe to secure access for eligible patients quickly.\n\n\nIn Europe, KALYDECO® (ivacaftor) is already approved for the treatment of people with CF ages 18 and older with the R117H mutation, and children 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, S1251N, S1255P, S549N or S549R.\n\n\nAbout Cystic Fibrosis\n\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,...

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