Phase III ARRIVAL data on Kalydeco in cystic fibrosis published in The Lancet Respiratory Medicine.- Vertex Pharma.

Vertex Pharmaceuticals announced data from the ongoing Phase III, open-label ARRIVAL study and published online in The Lancet Respiratory Medicine which shows that treatment with Kalydeco (ivacaftor) in children with cystic fibrosis (CF) revealed that ivacaftor was generally safe and well tolerated in children aged 12 to 24 months for up to 24 weeks and was associated with rapid and sustained reductions in sweat chloride concentrations. Improvements in biomarkers of pancreatic function suggest that ivacaftor preserves exocrine pancreatic function if started early.

The study is continuing in infants younger than 12 months. The data suggests the potential to preserve pancreatic function and modify the course of CF beginning in children as young as one year of age. In addition, final annual analyses of the completed, five-year, post-approval observational safety study of Kalydeco show that patients taking Kalydeco had lower risk of death, transplantation, hospitalization and pulmonary exacerbations compared to patients who were matched on age, gender and genotype class who did not receive Kalydeco. Together, these studies provide further support for the benefit of both early and long-term treatment with CFTR modulators. Data were presented at the 41st European Cystic Fibrosis Conference.

See: Ivacaftor treatment of cystic fibrosis in children aged 12 to <24 months and with a CFTR gating mutation (ARRIVAL): a phase III single-arm study