Tezacaftor

[3][4][5] The combination of tezacaftor with ivacaftor (brand name Symdeko) was approved for medical use in the United States in February 2018,[6][1] and in Canada in June 2018.

[8] The combination of tezacaftor with elexacaftor and ivacaftor (brand name Trikafta) was approved for medical use in the United States in October 2019,[9][10][11] and in Canada in June 2021.

[14] Tezacaftor acts as a corrector to help the folding and presentation of the CFTR protein to the cell surface, which improves its function for individuals with a F508del mutation.

[18] The combination of drugs was efficacious in participants who had cystic fibrosis with the Phe508del mutation and the adverse effects in both treatment groups were similar.

[8] The FDA and the EMA granted the application for elexacaftor, tezacaftor, and ivacaftor combination therapy an orphan drug designation.