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 2014 UK Cystic Fibrosis Registry Report, there are 2,834 people in England, 243 people in Scotland, 118 people in Wales and 101 people in Northern Ireland who could benefit from Orkambi.
- Orkambi has been licensed for use in the UK for people with CF over the age of two who have two copies of the F508del mutation. Despite this, the National Institute for Health and Care Excellence (NICE) has rejected its use on the NHS.
- In May 2016, the Scottish Medicines Consortium (SMC) noted that Orkambi was a beneficial treatment.In June 2016, NICE recognised Orkambi as an important treatment.
- Both the SMC and NICE were unable to recommend Orkambi for general use within the NHS/HSCNI on grounds of cost-effectiveness and a lack of long-term data.
- Orkambi is currently only prescribed to people on compassionate grounds. Compassionate use means that Vertex Pharmaceuticals provide the drug to people who fulfill a number of criteria. Contact your clinician for more information on compassionate use.
- Orkambi was licensed for 6-11 year olds by the European Medicines Agency in Europe in January 2018, and was again licensed for 2-5 year olds in January 2019.
- In August 2019 the Scottish Medicine Consortium (SMC) announced it was not able to recommend Orkambi (and similar drug, Symkevi) for routine use by NHS Scotland, adding that the company had failed to justify the price they were offering. Applications to use Orkambi in Scotland can still be made on an individual, case-by-case basis, under the PACS Tier 2 system and on compassionate grounds.
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.