Elexacaftor(ELX)/tezacaftor(TEZ)/ivacaftor(IVA) is a triple combination drug that is under investigation in a combination regime with ivacaftor for cystic fibrosis (CF) patients without an F508del mutation and with an ELX/TEZ/IVA-responsive mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. CF is an inherited disease, caused by CFTR mutations in the DNA, that progressively affects the lungs and digestive system causing breathing difficulties and problems absorbing nutrients from food. There are currently few licensed medications that treat patients with non-F508del, but ELX/TEZ/IVA responsive mutations in CF, resulting in an unmet need for these patients.
Elexacaftor/tezacaftor/ivacaftor and ivacaftor for treating cystic fibrosis without an F508del mutation and with an ELX/TEZ/IVA-responsive mutation in the CFTR gene in patients aged 6 years and older
Elexacaftor(ELX)/tezacaftor(TEZ)/ivacaftor(IVA) is a triple combination drug that is under investigation in a combination regime with ivacaftor for cystic fibrosis (CF) patients without an F508del mutation and with an ELX/TEZ/IVA-responsive mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. CF is an inherited disease, caused by CFTR mutations in the DNA, that progressively affects the lungs and digestive system causing breathing difficulties and problems absorbing nutrients from food.
Interventions:
Ivacaftor (Kalydeco; VX-770)
, Kaftrio (fixed dose combination: Elexacaftor; Tezacaftor; Ivacaftor)
Indications:
Cystic fibrosis
Year:
2023