Why is it prescribed?
Tezacaftor is used in combination with ivacaftor for the treatment of cystic fibrosis in patients aged 12 years and older:
- who are homozygous for the F508del mutation OR
- who are heterozygous for the F508del mutation and have one of the following mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene: P67L, D110H, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272-26A→G, and 3849+10kbC→T.
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Various mutations of the cystic fibrosis gene block the "gates" that allow the flow of salt across the cell membranes. This causes mucus to become thick and clog the lungs. Tezacaftor is a "cystic fibrosis transmembrane conductance regulator (CFTC) corrector". The CFTR protein is a channel at the surface of the cells that allows the movement of particles such as chloride in and out of the cell, contributing to salt and water balance. Tezacaftor corrects the positioning of the CFTR protein on the cell surface to permit proper channel formation and improved flow of water and salts across the cell membrane. The thick mucus is reduced and cystic fibrosis symptoms lessen.