elexacaftor

Elexacaftor is a small molecule cystic fibrosis transmembrane conductance regulator (CFTR) corrector developed for the treatment of cystic fibrosis. It is used in combination with tezacaftor and ivacaftor as part of the fixed-dose combination products Trikafta and Kaftrio. Elexacaftor works by modulating the CFTR protein to facilitate its trafficking to the cell surface and incorporation into the cell membrane, thereby increasing mature CFTR protein at the cell surface and improving ion transport. This mechanism helps reduce symptoms associated with cystic fibrosis in patients who have at least one F508del mutation or another responsive mutation in their CFTR gene[4][6][8]. The triple-combination therapy provides a synergistic effect beyond dual corrector/potentiator regimens. Developed by Vertex Pharmaceuticals.

Brand names

TrikaftaKaftrio

Other names

ELXELX/TEZ/IVAVX-445/TEZ/IVA