Medical Uses
Kaftrio (elexacaftor/tezacaftor/ivacaftor), from Vertex Pharmaceuticals, is the EU brand name for the triple-combination CFTR modulator therapy (marketed as Trikafta in the US) indicated for the treatment of cystic fibrosis (CF) in patients with at least one F508del mutation in the CFTR gene, or other responsive mutations, as determined by an approved genotype.
Kaftrio combines three CFTR modulators: elexacaftor and tezacaftor help the defective CFTR protein fold and traffic correctly to the cell surface, while ivacaftor acts as a potentiator, keeping the CFTR channel open longer to improve chloride transport. Together they improve the function of the underlying defective protein responsible for cystic fibrosis.
