Elexacaftor

Elexacaftor is a medication that acts as cystic fibrosis transmembrane conductance regulator corrector, which means that it helps the CFTR protein fold correctly, increasing the amount of CFTR protein sent to the cell surface. It also appears to work as a CFTR potentiator, which means that it helps open any CFTR channel that have made its way onto the cell surface.
It is available in a single pill with ivacaftor and tezacaftor; the fixed-dose combination, elexacaftor/tezacaftor/ivacaftor, is used to treat people with cystic fibrosis who are homozygous for the f508del mutation. This combination was approved for medical use in the United States in 2019.
The fixed-dose combination elexacaftor/tezacaftor/ivacaftor was approved for medical use in the European Union in August 2020, for the treatment of cystic fibrosis.