Icenticaftor

Icenticaftor is an orally active small molecule that acts as a potentiator of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. It works by keeping the CFTR channel open longer to allow improved chloride ion flow across epithelial cell membranes. This mechanism targets defective CFTR function seen in diseases such as cystic fibrosis and chronic obstructive pulmonary disease (COPD), particularly in patients with certain CFTR mutations or chronic bronchitis phenotypes. Icenticaftor was developed by Novartis and has been evaluated in clinical trials for both COPD and cystic fibrosis. While it showed some improvements in secondary endpoints like cough reduction and lung function measures, development for COPD has been discontinued after phase II trials; development for cystic fibrosis and bronchiectasis reached phase II[1][3][4][6][7].

Other names

icenticaftorQBW251QBW-251QBW 251NVP-QBW-251NVP-QBW251NVP-QBW 251