1 Recommendations

1 Recommendations

1.1 Lumacaftor–ivacaftor is not recommended, within its marketing authorisation, for treating cystic fibrosis in people 12 years and older who are homozygous for the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

1.2 This guidance is not intended to affect the position of patients whose treatment with lumacaftor–ivacaftor was started within the NHS before this guidance was published. Treatment of those patients may continue without change to whatever funding arrangements were in place for them before this guidance was published until they and their NHS clinician consider it appropriate to stop. For children and young people, this decision should be made jointly by the clinician and the child or young person or the child or young person's parents or carers.

  • National Institute for Health and Care Excellence (NICE)