Medical Uses
Lucaftor (lumacaftor/ivacaftor) is a fixed-dose combination CFTR modulator therapy indicated for the treatment of cystic fibrosis (CF) in patients who are homozygous for the F508del mutation in the CFTR gene.
Lumacaftor helps correct the misfolded CFTR protein so it can move to the cell surface, while ivacaftor acts as a potentiator, helping the CFTR protein at the cell surface function more effectively — together improving chloride channel activity and helping reduce the buildup of thick, sticky mucus characteristic of CF.
