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QAU145 is a small molecule drug developed by Novartis for the treatment of cystic fibrosis. It acts as an inhibitor of epithelial sodium channel (ENaC)-mediated sodium transport in airway epithelial cells, aiming to reduce excessive and unregulated sodium absorption that characterizes cystic fibrosis airways. By inhibiting ENaC, QAU145 is intended to increase airway surface hydration and improve mucociliary clearance. The drug was administered via intranasal spray in clinical trials and demonstrated a dose-dependent pharmacodynamic effect on nasal potential difference, indicating its activity on ion transport in the respiratory epithelium. Clinical studies have shown that single intranasal doses were generally safe and well tolerated[2][4][5][7].
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