European CHMP recommends approval of elexacaftor / tezacaftor / ivacaftor (Kaftrio)

The license is for the treatment of cystic fibrosis in patients aged 12 years and older who are homozygous for the F508del mutation or heterozygous for the F508del in the cystic fibrosis transmembrane conductance regulator (CFTR) gene with a minimal function mutation.

SPS commentary:

In a related press release the European Medicines Agency state this is the first triple combination therapy for the treatment of cystic fibrosis. Elexacaftor, tezacaftor and ivacaftor work by increasing CFTR protein and improving its function.


European Medicines Agency

Resource links:

Press release