Cystic Fibrosis study offers potential to modify the course of CF in children
Vertex Pharmaceuticals Incorporated announced positive results from an open-label Phase III study of Kalydeco (ivacaftor) in children with cystic fibrosis (CF) ages 1 to 2 years who have one of 10 mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
The ARRIVAL study met its primary endpoint of safety, showing that ivacaftor was generally well tolerated, and safety data were consistent with those seen in previous Phase III studies of ivacaftor in children ages 2 to 5 years and 6 to 11 years. There was also a substantial improvement in sweat chloride, a secondary endpoint, as well as in multiple exploratory endpoints evaluating pancreatic function. These data suggest the potential to modify the course of CF in children as young as one year of age. The study is ongoing in infants younger than one year old.
Based on these results, Vertex plans to submit applications for ivacaftor in children ages 1 to 2 years to the FDA and the European Medicines Agency (EMA) in the first quarter of 2018.
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