Ivacaftor–tezacaftor–elexacaftor (IVA–TEZ–ELX) plus ivacaftor (IVA) alone is recommended within its marketing authorisation, as an option for treating cystic fibrosis in people 2 years and over who have at least 1 F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
Tezacaftor–ivacaftor (TEZ–IVA) plus IVA alone is recommended, within its marketing authorisation, for treating cystic fibrosis in people 6 years and over who have:
2 copies of the CFTR gene with F508del mutations, or
a copy of the CFTR gene with an F508del mutation and a copy of the CFTR gene with 1 of the mutations listed in section 2.2.
Lumacaftor–ivacaftor (LUM–IVA) is recommended, within its marketing authorisation, for treating cystic fibrosis in people 1 year and over who have 2 copies of the CFTR gene with F508del mutations.
IVA–TEZ–ELX, TEZ–IVA and LUM–IVA are only recommended if the company provides them according to the commercial arrangement.
Why the committee made these recommendations
Cystic fibrosis causes a range of challenging symptoms that affect the lungs, digestive system and liver. People with cystic fibrosis have a shortened life expectancy and a greatly reduced quality of life. Usual treatment aims to manage the symptoms and includes several intensive treatments and physical therapies. Treatment is very physically demanding and time consuming for people with cystic fibrosis and their families and carers.
Clinical trial evidence shows that IVA–TEZ–ELX improves lung function, growth and weight gain and reduces the number of lung infections more than standard treatment. It is likely that these benefits last while people are having treatment.
Clinical trial evidence shows that TEZ–IVA and LUM–IVA also improve lung function, growth and weight gain and reduce the number of lung infections more than standard treatment. But the short- and long-term improvements are smaller than with IVA–TEZ–ELX.
When considering the condition's severity, and its effect on quality and length of life, the most likely cost-effectiveness estimates for IVA–TEZ–ELX, LUM–IVA and TEZ–IVA are within what NICE considers an acceptable use of NHS resources. So, they are recommended.