vanzacaftor

Vanzacaftor is a small molecule drug developed as a cystic fibrosis transmembrane conductance regulator (CFTR) corrector. It is used as part of a fixed-dose combination with tezacaftor and deutivacaftor (marketed as Alyftrek) for the treatment of cystic fibrosis in patients aged 6 years and older who have at least one F508del mutation or another responsive mutation in the CFTR gene. Vanzacaftor acts by binding to misfolded CFTR protein, stabilizing its structure, and ensuring it reaches the cell surface where it can function correctly, thereby helping restore chloride and water transport across cell membranes[4][5][6][7]. The combination therapy improves cellular processing and trafficking of CFTR protein (vanzacaftor/tezacaftor), while deutivacaftor acts as a potentiator to enhance channel gating activity[8].

Other names

vanzacaftor