FDA approves Kalydeco to treat infants with CFTR gene cystic fibrosis

Vertex Pharmaceuticals Incorporated announced the FDA approved Kalydeco (ivacaftor) for use in children with cystic fibrosis (CF) ages six months to less than 12 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. Kalydeco is already approved in the U.S., Canada and EU for the treatment of CF in patients ages 12 months and older.

This FDA approval is based on data from a 24-week Phase III open-label safety cohort (ARRIVAL) of 11 children with CF aged six months to less than 12 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 similar to 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 therapy due to adverse events. The most common adverse events ( greater than 30%) were cough (64%), nasal congestion (36%) and rhinorrhea (36%). Three serious adverse events, all considered unrelated to study treatment by the investigators, were observed in three patients. Mean baseline sweat chloride for the children in this cohort was 101.5 mmol/L (n=11). Following 24 weeks of treatment withKalydeco , the mean sweat chloride level was 43.1 mmol/L (n=6). In the six subjects with paired sweat chloride samples at baseline and week 24, there was a mean absolute change of -58.6 mmol/L (95% CI; -75.9, -41.3). Results of this study were presented at the 32nd Annual North American Cystic Fibrosis Conference in October 2018.

Read more on the Cystic Fibrosis Knowledge Centre.