MECHANISM OF ACTION

Targeting responsive CFTR mutations increases CFTR activity1,2

DEFECTIVE CFTR PROTEINS

Overall CFTR activity may be decreased by

  • Impaired cellular processing and trafficking1

and/
or

  • Reduced channel-open probability1

and/
or

SPECIFIC RESPONSIVE PROTEINS

Overall CFTR activity is increased by

tcus_image

 

  • Improved cellular processing and trafficking (elexacaftor and tezacaftor) and potentiated channel-open probability (ivacaftor)1,2

 

TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) is approved for patients with CF aged 6 years and older who have at least one F508del mutation in the CFTR gene or a mutation in the CFTR gene that is responsive based on in vitro data2


CF, cystic fibrosis; CFTR, cystic fibrosis transmembrane conductance regulator.