FDA approves Kalydeco to treat cystic fibrosis in children aged 12 to <24 months.- Vertex Pharma.

Vertex Pharmaceuticals Incorporated announced the FDA approved Kalydeco (ivacaftor) to include use in children with cystic fibrosis (CF) ages 12 to <24 months who have at least one mutation in their cystic fibrosis transmembrane conductance regulator cftr gene that is responsive to kalydeco based on clinical and or in vitro assay data.>

This FDA approval is based on data from the ongoing Phase III open-label safety study (ARRIVAL) of 25 children with CF aged 12 to <24 months who have one of 10 mutations in the cftr gene g551d g178r s549n s549r g551s g1244e s1251n s1255p g1349d or r117h. the study demonstrated a safety profile consistent with that observed in previous phase iii studies of older children and adults most adverse events were mild or moderate in severity and no patient discontinued due to adverse events. two patients had elevated liver enzymes greater than eight times the upper limit of normal but continued to receive kalydeco after a dose interruption. the most common adverse events 30 were cough 74 pyrexia 37 elevated aspartate aminotransferase 37 elevated alanine aminotransferase 32 and runny nose 32. four serious adverse events were observed in two patients.>

Mean baseline sweat chloride for the children in this study was 104.1 mmol/L (n=14). Following 24 weeks of treatment with Kalydeco, the mean sweat chloride level was 33.8 mmol/L (n=14). In the 10 subjects with paired sweat chloride samples at baseline and week 24, there was a mean absolute change of -73.5 mmol/L. These data were presented at the 41st European Cystic Fibrosis Society (ECFS) Conference in June 2018 and published in The Lancet Respiratory Medicine (Volume 6, No 7, July 2018).

See-

"Ivacaftor treatment of cystic fibrosis in children aged 12 to <24 months and with a cftr gating mutation arrival: a phase 3 single-arm study- margaret rosenfeld et al. the lancet respiratory medicine 6 june 2018 doi: https: doi.org 10.1016 s2213-26001830202-9>