NICE in final guidance does not recommend Orkambi for cystic fibrosis in people with two copies of the F508del mutation. Vertex Pharma.

The National Institute for Health and Care Excellence (NICE) has issued a final appraisal determination rejecting cystic fibrosis drug, Orkambi (lumacaftor/ivacaftor), which is developed by Vertex Pharmaceuticals. Despite describing it as a “valuable new therapy for managing cystic fibrosis”, the drug will not be made available to patients on the NHS in England.

The drug is indicated for people with cystic fibrosis aged 12 and over who have two copies of the F508del mutation. Vertex describes Orkambi as the “first medicine to treat the underlying cause of cystic fibrosis in people with two copies of the F508del mutation.”

NICE did not recommend the drug in initial draft guidance. At the time, the independent appraisal committee concluded that “the cost of Orkambi was considerably higher than the current standard of care and it could not be considered a cost effective use of NHS resources.” They indicated that its cost of £104,000 per year per patient was excessive and that this cost would be exacerbated by most patients remaining on currently-available treatments while taking Orkambi.