Landmark study involving babies in Ireland supports use of Cystic Fibrosis drug in infants from four weeks of age

By Danielle Norton April 11, 2024 Updated May 16, 2024 4 min read

REPRO FREE - NO REPRO FEE 11/4/2024 Pictured are siblings Kara (aged 5) and Isaac Moss (aged 2), with their parents Debbie and Wayne Moss, who participated in a landmark study which supports use of a ground-breaking Cystic Fibrosis drug in infants from four weeks of age. © Patrick Bolger The research by RCSI University of Medicine and Health Sciences and Children’s Health Ireland (CHI) is published in the in the Journal of Cystic Fibrosis. Kara was part of the earlier phase of the study that paved the approval of the drug in older infants and led to the latest trial that Isaac took part in. Isaac was the first baby with Cystic Fibrosis in the world to be diagnosed from birth and enrolled directly onto a trial of these treatments using Ivacaftor (Kalydeko), the first drug designed to treat the basic defect in Cystic Fibrosis. Cystic Fibrosis experts predict that the earlier treatments can begin, the more likely that progression of the condition can be slowed down or halted in children.

A Cystic Fibrosis drug targeting the basic defect that causes the condition has been shown to be safe and effective in newborns aged four weeks and above, new research involving RCSI University of Medicine and Health Sciences and Children’s Health Ireland has found.

The finding is described as a ‘huge moment’ for Cystic Fibrosis by one of the lead researchers. The study included the first baby in the world with Cystic Fibrosis to be diagnosed from birth and enrolled directly onto a trial of this sort.