Vertex announced that the Food and Drug Administration (FDA) has approved Kalydeco (ivacaftor) to include use in pediatric patients with cystic fibrosis (CF) ages 12–<24 months who have ≥1 mutation in their cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to Kalydeco based on clinical and/or in vitro assay data.

The approval was supported by data from ARRIVAL (N=25), an ongoing Phase 3 open-label safety study in children with CF aged 12 to <24 months with 1 of 10 CFTR gene mutations (G551DG178R, S549NS549R, G551S, G1244E, S1251N, S1255P, G1349D or R117H). The data showed a safety profile comparable to that seen in previous Phase 3 studies of older children and adults. There were no treatment discontinuations due to adverse events and most events were mild or moderate in severity. Cough was the most common adverse event reported, followed by pyrexia, elevated AST/ALT, and runny nose. 

Additional data demonstrated a decrease in the mean baseline sweat chloride level after 24 weeks of treatment (104.1mmol/L to 33.8mmol/L). Among the 10 patients with paired sweat chloride samples at baseline and at week 24, the mean absolute change was -73.5mmol/L. 

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Kalydeco, a CFTR potentiator, is already indicated to treat CF in patients aged ≥2 years who have 1 of 38 ivacaftor-responsive mutations in the CFTR gene based on clinical and/or in vitro assay data. It is available as 150mg tablets for patients aged ≥6 years, and as 50mg and 75mg oral granules for patients aged 12 months to <6 years. 

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