FDL169

FDL169 is an investigational small molecule drug developed as a CFTR corrector for the treatment of cystic fibrosis (CF), specifically targeting patients who are homozygous for the F508del mutation in the CFTR gene. The drug is designed to bind to and assist in the proper folding of the defective CFTR protein caused by this mutation, thereby increasing its presence at the cell membrane and restoring chloride channel function. This mechanism aims to improve salt transport across epithelial cells and alleviate symptoms associated with cystic fibrosis. Multiple Phase 1 clinical trials have evaluated its safety, pharmacokinetics, and pharmacodynamics in both healthy volunteers and people with cystic fibrosis; however, it has not been approved by any regulatory authority for marketing[3][5][6][7].

Other names

CFTR correctorCFTR-corrector