Drug intelligence / Profile preview

elexacaftor + tezacaftor + ivacaftor

Development stage
Approved
Lead developer
Vertex
Modality
Small Molecules
Administration
Oral
01

Overview

Elexacaftor + tezacaftor + ivacaftor is a fixed-dose combination of three small-molecule drugs used to treat cystic fibrosis (CF) in patients with at least one copy of the F508del mutation or another responsive mutation in the CFTR gene. Elexacaftor and tezacaftor are classified as CFTR correctors; they work by facilitating proper folding and trafficking of the CFTR protein to increase its presence on the cell surface. Ivacaftor is a CFTR potentiator that increases chloride channel opening probability, enhancing ion transport across epithelial cells. The combination improves chloride and sodium ion transport, reducing thick mucus buildup characteristic of cystic fibrosis. This therapy is indicated for adults and children aged 2 years and older with eligible mutations[1][2][3][5][6].

Brand names
TrikaftaKaftrio
Other names
ELX/TEZ/IVAelexacaftor-tezacaftor-ivacaftor
02

Targets

CFTR (Cystic fibrosis transmembrane conductance regulator)

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