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Vertex Says Phase 3 Study Of KALYDECO Met Primary Endpoint

Vertex Pharmaceuticals Inc. (VRTX) announced positive results from an open-label Phase 3 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 or 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 3 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.

The 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 U.S. Food and Drug Administration and the European Medicines Agency or EMA in the first quarter of 2018.

KALYDECO is currently approved by the FDA for the treatment of CF in patients ages 2 and older who have one of 38 ivacaftor-responsive mutations in the CFTR gene.

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