New cystic fibrosis combination drug not recommended for NHS use

Orkambi (lumacaftor/ivacaftor), a new gene-specific medication for cystic fibrosis, has been rejected by NICE on cost-effectiveness grounds.

Orkambi (lumacaftor/ivacaftor) targets the CFTR chloride channel to treat the characteristic respiratory problems of cystic fibrosis. | Image provided by Vertex Pharmaceuticals
Orkambi (lumacaftor/ivacaftor) targets the CFTR chloride channel to treat the characteristic respiratory problems of cystic fibrosis. | Image provided by Vertex Pharmaceuticals

NICE concluded that Orkambi, a combination of 200mg lumacaftor and 125mg ivacaftor, "has the potential to ease the treatment burden of cystic fibrosis by reducing the number of pulmonary exacerbations", but described the drug's cost-effectiveness as "considerably higher than what is normally considered a cost-effective use of NHS resources", and has not recommended it for use across the NHS.

Further information
View Orkambi drug record
NICE technology appraisal TA398

In May, Orkambi was turned down by the Scottish Medicines Consortium, which said the drug's manufacturer, Vertex, had not provided "a sufficiently robust clinical and economic analysis" for use of the treatment within NHS Scotland.

Vertex has now submitted a patient access scheme for Orkambi in England and plans to propose a similar scheme for Scotland. Talks are also underway with a view to making Orkambi available to patients in Northern Ireland and Wales.

Orkambi is indicated for the treatment of cystic fibrosis in patients from the age of 12 years who are homozygous for the F508del mutation in the gene encoding the CFTR chloride channel.

F508del is the most common causative cystic fibrosis mutation. Lumacaftor, a CFTR corrector, increases the amount of CFTR at cell surfaces, and ivacaftor, a CFTR potentiator, enhances gating of the channels.

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