What is Orkambi?
Orkambi is a combination medicine, made up of ivacaftor and lumacaftor. The treatment has been shown to be effective in people with CF with two copies of the F508del mutation, which around 50% of people with CF in the UK have.
Is Orkambi available in the UK?
Orkambi is available for use in the UK for people with CF over the age of two with two copies of the F508del mutation.
Managed access agreements to make Orkambi available on the NHS have now been agreed in England, Scotland, Wales and Northern Ireland. To find out more about these agreements, visit our FAQs page.
To find out whether you or your child might be eligible for a precision medicine, please speak to your CF team.
What effect does Orkambi have?
96-week data shows that Orkambi can slow decline in lung function - the main cause of death among people with cystic fibrosis - by 42%.
How is the Trust supporting the process?
The UK CF Registry, which is maintained by the Trust, is playing a vital role in supporting these agreements by collecting data on the health outcomes of individuals taking Orkambi and other recently licensed drugs.
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