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Cystic Fibrosis Trust


Orkambi (ivacaftor/lumacaftor) is the second precision medicine to be licensed for use in the UK by people with cystic fibrosis (CF), although it is not currently provided on the NHS. It is provided by Vertex Pharmaceuticals only in rare cases on compassionate grounds for those who fulfill a number of criteria.

What is Orkambi?

Orkambi is the second such drug to be licensed for use in people with cystic fibrosis, the first was Kalydeco.

  • Orkambi treats the F508del mutation, which around 50% of people with CF in the UK have.
  • Orkambi is a combination medicine, made up of ivacaftor and lumacaftor. Lumacaftor helps get more proteins to the surface of cells in the body, and ivacaftor helps the chloride channels in the cells to operate more effectively. The combination of these two things helps to keep a healthy balance of salt and water in the organs – particularly the lungs. Orkambi is manufactured by Vertex Pharmaceuticals.

Is Orkambi available in the UK?

According to the 2017 UK Cystic Fibrosis Registry Report, there are 3,968 people in England, 350 people in Scotland, 169 people in Wales and 156 people in Northern Ireland who could benefit from Orkambi.

Orkambi is licensed for use in the UK for people with CF over the age of two who have two copies of the F508del mutation, and in September 2019 the Scottish Government announced a five-year deal giving access to the drug for everyone with CF in Scotland who can benefit from it.

However, despite recognising Orkambi as an important treatment, both the Scottish Medicines Consortium (SMC) and English watchdog, the National Institute for Health and Care Excellence (NICE), have not been able to recommend Orkambi for use within the NHS on grounds of cost-effectiveness and a lack of long-term data. In England, Wales and Northern Ireland the drug is currently only prescribed to people on compassionate grounds and through limited clinical trial access.

Compassionate use means that Vertex Pharmaceuticals provide the drug to people who fulfil a number of criteria. 

To find out whether you or your child might be eligible for a precision medicine, whether in Scotland or through compassionate use, 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%. This data was unavailable to NICE when they appraised Orkambi.

Why is Orkambi not available to everyone who needs it?

The processes involved in making drugs available to the people they could help are complicated. Creating and testing a drug like Orkambi is very expensive and a lack of long-term data can make it hard for NICE and the SMC, the bodies that appraises these drugs for use in the NHS/HSCNI, to accurately assess their cost-effectiveness in order to make them available.

What are we doing about it?

Stay up to date with Stopping the Clock, our dedicated campaign to put pressure on the Government, NHS and pharmaceutical companies to ensure these drugs reach the people who need them without delay, join the campaign and get involved yourself by taking part in campaigning in your area.

Stopping the Clock

Find out more about our campaign to ensure that people with CF across the UK can have access to innovative precision medicines.

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