Kaftrio (Elexacaftor/tezacaftor/ivacaftor) and ivacaftor for treating cystic fibrosis in children (2 to 5 years) with at least one F508del mutation in the CFTR gene

Kaftrio (Elexacaftor(ELX)/tezacaftor(TEZ)/ivacaftor(IVA)) is a triple combination drug that is licensed for patients with cystic fibrosis (CF) aged 6 years and older and is being investigated for use in patients aged 2 to 5 years. CF is an inherited disease that progressively effects the lungs and digestive system causing breathing difficulties and problems absorbing nutrients from food. There are currently no licensed medicines or combinations that act on all variations of F508del mutations that cause the symptoms and damage in CF for patients under 6 years old. Existing treatments are limited to certain populations leaving others without treatment options. Kaftrio would provide an option to those previously excluded from these treatments.