Medical Uses
Symkevi (tezacaftor/ivacaftor), from Vertex Pharmaceuticals, is indicated for the treatment of cystic fibrosis (CF) in patients age 6 and older who are homozygous for the F508del mutation, or who have at least one mutation responsive to tezacaftor/ivacaftor, in combination with ivacaftor.
Tezacaftor corrects the misfolded CFTR protein so it can move to the cell surface, while ivacaftor potentiates the CFTR protein already at the cell surface, together improving chloride channel function.
