GLPG2222 + ivacaftor

GLPG2222 + ivacaftor is an **investigational combination therapy for cystic fibrosis**. GLPG2222 (also known as ABBV-2222) is a small molecule **CFTR corrector** developed to improve the folding and trafficking of the F508del mutant CFTR protein to the cell surface. Ivacaftor is a well-established **CFTR potentiator** that increases the channel opening probability of CFTR protein at the cell membrane, primarily used in patients with gating mutations (e.g., G551D). The combination is designed for patients who are compound heterozygotes for a gating mutation (Class III) and F508del (Class II), with the intention to enhance CFTR function by both increasing the presence of CFTR at the cell surface (corrector effect from GLPG2222) and enhancing its activity (potentiator effect from ivacaftor). This combination has been tested in Phase II clinical trials (ALBATROSS study), showing a dose-dependent decrease in sweat chloride and a mild increase in FEV1, indicating on-target activity, and was well tolerated in CF patients already receiving stable ivacaftor therapy[1][3][5].

Brand names

Kalydeco

Other names

GLPG2222 + KalydecoABBV-2222 + ivacaftor