What you need to know about Orkambi
Precision medicines for cystic fibrosis treat the underlying genetic mutation that causes the condition, whereas traditional medicines treat only the symptoms. This means that precision medicines can have a huge impact on quality of life by reducing the amount of time spent in hospital and slowing the decline in lung function experienced by people with cystic fibrosis.
There are many different mutations that can cause cystic fibrosis, and each person with the condition will have two of these. They can be the same, or two different mutations in one individual. Precision medicines are so called because they will treat only certain mutations, rather than a symptom, such as pancreatic insufficiency, which could affect everyone with cystic fibrosis. These drugs are part of a wider move towards personalised healthcare.
The first precision medicine for cystic fibrosis was Kalydeco. Orkambi is the second such drug to be licensed for use in people with cystic fibrosis.
What is Orkambi?
Orkambi treats the F508del mutation, which around 40% of people with cystic fibrosis in the UK have. It is a combination medicine, made up of Kalydeco (Ivacaftor) and Lumacaftor. The 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.
Who does Orkambi work on?
Orkambi is effective in treating the 40% of the UK population of people with cystic fibrosis who have two copies of the F508del mutation.
Who is currently receiving Orkambi in the UK?
Despite having been licensed for use in the UK for people with CF over the age of 12 who have two copies of the F508del mutation, NICE rejected its use on the NHS. It is currently only being prescribed to people on compassionate grounds from just 25 CF centres. Compassionate use means that Vertex Pharmceuticals (the manufacturer of Orkambi) has an arrangement in place allowing people who are seriously ill to take the drug free of charge. There are certain requirements that have to be met for compassionate use to be considered – if you would like more information please contact your clinician.
Accordingly to the 2014 UK Cystic Fibrosis Registry Report, there are 2,789 people in England, 236 people in Scotland, 113 people in Wales and 111 people in Northern Ireland who could stand to benefit from Orkambi.
What effect does Orkambi have?
Recent data from a 96-week study has demonstrated that Orkambi can slow down the decline in lung function experienced by people with cystic fibrosis by an average of 42%. This decline of lung function is the main cause of death in people with cystic fibrosis and has a serious impact on quality of life and an individual’s ability to stay active and independent.
Why is it 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, the body that appraises these drugs for use in the NHS, to accurately assess their cost-effectiveness in order to make them available.
What is the Cystic Fibrosis Trust doing?
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.