deutivacaftor

Deutivacaftor is a small molecule drug classified as a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator. It is a deuterated analog of ivacaftor designed to increase the stability and duration of action in the body, allowing for once-daily dosing. Deutivacaftor works by increasing the open probability of functional CFTR chloride channels at the cell surface, thereby enhancing chloride transport in patients with certain CFTR mutations. It is primarily used as part of a fixed-dose combination with vanzacaftor and tezacaftor (marketed as Alyftrek) for the treatment of cystic fibrosis in patients aged six years and older who have at least one F508del mutation or another responsive mutation in the CFTR gene.

Brand names

Alyftrek

Other names

deuterated ivacaftor