Lumacaftor-Ivacaftor for Treating Cystic Fibrosis Homozygous for the F508del Mutation (TA398)

Technology Appraisal Guidance No. 398

Source: National Institute for Health and Care Excellence

1. Guidance

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.


The guidance shown above constitutes Section 1 of the full document. A copy of the full document and a summary of the evidence is available on the Internet at 

This guidance represents the view of the Institute which was arrived at after careful consideration of the available evidence. Health professionals are expected to fully take it into account when exercising their clinical judgement. This guidance does not, however, override the individual responsibility of health professionals to make appropriate decisions in the circumstances of the individual patient, in consultation with the patient and/or guardian or carer.

© Copyright National Institute for Health and Care Excellence. All rights reserved. This material may be freely reproduced for educational and not for profit purposes within the NHS. No reproduction by or for commercial organisations is permitted without the express written permission of the Institute.

Enquiries concerning the guidance should be addressed to: National Institute for Health and Care Excellence, MidCity Place, 71 High Holborn, London WC1V 6NA. email: [email protected]

Lumacaftor–ivacaftor for treating cystic fibrosis homozygous for the F508del mutation

Issue Date: July 2016


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