Kalydeco (ivacaftor) was shown to be safe and well-tolerated in 1- and 2-year-old children with cystic fibrosis, based on data from a Phase 3 trial, Vertex Pharmaceuticals announced.
Based on the positive interim results, Vertex said that in the first quarter of 2018, it would seek regulatory approval in the United States and Europe for the use of Kalydeco for treatment of CF in children ages 1 to 2.
“We know that cystic fibrosis is a progressive disease with organ damage already present at birth, so the earlier patients can begin treatment, the greater their potential for improved outcomes,” Jane Davies, MD, from the Royal Brompton Hospital and Imperial College London, and co-lead investigator of the ARRIVAL study, said in a press release.
“These results are incredibly exciting: they suggest that we can begin treating the underlying cause of cystic fibrosis with Kalydeco in children as young as one year of age,” Davies added.
