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Why is it prescribed?

Elexacaftor is used in combination with similar medications to treat cystic fibrosis (CF) in patients 12 years of age and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Products that have this ingredient include •Trikafta® •

See other drugs used in the treatment of •cystic fibrosis •

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. Elexacaftor 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. Elexacaftor 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.