tezacaftor

Tezacaftor is a small molecule drug used in the treatment of cystic fibrosis (CF), particularly in patients with homozygous or heterozygous F508del mutations in the CFTR gene. It acts as a cystic fibrosis transmembrane conductance regulator (CFTR) corrector, facilitating proper folding and trafficking of the CFTR protein to the cell surface. This improves chloride ion transport across epithelial cells and reduces thick mucus production characteristic of CF. Tezacaftor is typically administered in combination with ivacaftor (a CFTR potentiator) and sometimes elexacaftor for enhanced efficacy. The drug was developed by Vertex Pharmaceuticals and is marketed under brand names such as Symdeko (tezacaftor/ivacaftor) and Trikafta (elexacaftor/tezacaftor/ivacaftor)[3][7][6].

Brand names

SymdekoTrikafta