UK NICE does not recommend Orkambi (lumacaftor-ivacaftor) to treat cystic fibrosis F508del mutation- Vertex Pharma

NICE – The National Institute for Health and Care Excellence – has issued draft guidance which does not recommend Orkambi (lumacaftor-ivacaftor) marketed by Vertex Pharmaceuticals for treating cystic fibrosis. 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. There is currently no cure for cystic fibrosis and current treatments aim to manage individual symptoms.

Orkambi is licensed to treat people who have a specific genetic defect known as the F508del mutation. There are around 2,750 people in England with this genotype. It costs £104,000 per patient for every year of treatment. Most people suitable for Orkambi would also still remain on the current standard of care whilst taking it. The committee acknowledged that Orkambi does offer benefit because it reduces instances where people experience a sudden worsening of symptoms that requires hospitalisation. However, the benefits to lung function – the test to see how people with cystic fibrosis are improving overall – were modest.