The United States Food and Drug Administration (FDA) has approved United States-based Vertex Pharmaceuticals Incorporated's (Nasdaq:VRTX) KALYDECO (ivacaftor) intended for use in children with cystic fibrosis (CF) aged six months to less than 12 months who have at least one mutation in their cystic fibrosis transmembrane conductance regulator gene that is responsive to KALYDECO based on clinical and/or in vitro assay data, it was reported yesterday.
The product is already approved in the United States, Canada and European Union.
The approval is based on data from a 24-week Phase three 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 indicated a safety profile similar to that observed in previous Phase three 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 (up to 30%) were cough (64%), nasal congestion (36%) and rhinorrhoea (36%). Three serious adverse events, all considered unrelated to study treatment by the investigators, were observed in three patients.
Results of this study were presented at the 32nd Annual North American Cystic Fibrosis Conference in October 2018.
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