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Vertex Announces European Commission Approval for KALYDECO® (ivacaftor) for Children and Adolescents With Cystic Fibrosis Between the Ages of 6 Months and 18 Years With the R117H Mutation in the CFTR Gene
- KALYDECO® (ivacaftor) is the first and only approved medicine in Europe to treat the underlying cause of cystic fibrosis in patients with the R117H
About this update from Vertex Pharmaceuticals Incorporated
[{"type":"text","content":"\n- KALYDECO® (ivacaftor) is the first and only approved medicine in Europe to treat the underlying cause of cystic fibrosis in patients with the R117H mutation, the most common residual function mutation, in children as young as 6 months of age -\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) to include the treatment of children and adolescents with cystic fibrosis (CF), ages 6 months and older and weighing at least 5 kg, who have the R117H mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene – the most common residual function mutation underlying CF.\n\n\n“A little over eight years ago, KALYDECO® was approved as the first and only medicine to treat the underlying cause of cystic fibrosis in patients with specific mutations,” said Reshma Kewalramani, M.D., Chief Executive Officer and President at Vertex. “Since then, it’s been our goal to ensure that as many people with CF as possible are able to benefit from our treatments, and today’s label extension means that approximately 500 young patients in Europe, who have long awaited a treatment option, are now eligible for KALYDECO®.”\n\n\nNow approved, KALYDECO® (ivacaftor) will be immediately available to additional eligible patients in Germany and shortly in countries where respective long-term reimbursement agreements have been previously secured. Vertex will work closely with all other relevant government authorities to secure access for eligible patients as quickly as possible.\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 in infants 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, 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. Children must inherit ...