elexacaftor

Elexacaftor is a medication used to treat cystic fibrosis (CF). It is a cystic fibrosis transmembrane conductance regulator (CFTR) modulator that is marketed in combination with tezacaftor and ivacaftor as a single tablet under the brand names Trikafta in the United States and Kaftrio in the European Union. The triple-combination therapy was developed by Vertex Pharmaceuticals.

Elexacaftor acts as a CFTR corrector, promoting proper folding and trafficking of the CFTR protein to the cell surface. When used with tezacaftor, another corrector, and ivacaftor, a CFTR potentiator, the combination increases the amount of functional CFTR at the cell surface and enhances channel activity, leading to improved chloride transport and mucociliary clearance in the lungs.

In regulatory terms, Trikafta/Kaftrio is approved for people with CF who have at least one F508del mutation, with age and mutational eligibility varying by jurisdiction. In the United States, it has been approved for certain pediatric and adult patients as well as for younger children in some regions.

Common adverse effects include headache, nausea, diarrhea, rash, and nasopharyngitis. Elevations in liver enzymes have been reported, and liver function tests should be monitored. The combination is metabolized by and can interact with strong CYP3A inhibitors or inducers, so concomitant medications should be reviewed for drug interactions.

Elexacaftor, tezacaftor, and ivacaftor represent a major advancement in CFTR modulator therapy, offering substantial improvements in lung function and quality of life for many patients with CF.