Trial proves safety and efficacy of cystic fibrosis drug

Ivacaftor is the first drug designed to treat the basic defect in children born with the condition

Max Ryan

April 12, 2024

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  • 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 Irish research has found. 
    The study, carried out by the RCSI University of Medicine and Health Sciences and Children’s Health Ireland, included the first baby in the world with cystic fibrosis to be diagnosed from birth and enrolled directly onto a trial of this sort.
    The drug, ivacaftor, is the first drug designed to treat the basic defect in cystic fibrosis. It was originally approved for adults, then sequentially over several years for older and younger children. Currently, it is approved for babies aged four months and older, however, this new research suggests that it is safe and effective for babies as young as four weeks of age.
    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, and this is the focus of several international research studies led by RCSI and Children’s Health Ireland.
    It is hoped that the findings of this study could pave the way for eligible newborns to start treatment on the medicine at the time of diagnosis (typically at newborn screening) rather than having to wait until they are four months old.
    “This is a huge moment in Cystic Fibrosis,” said Paul McNally, associate professor of paediatrics at the RCSI and consultant in respiratory medicine at CHI.
    Prof McNally is one of the authors of the new study, which was published in the Journal of Cystic Fibrosis.
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