What is a precision medicine?
- Whilst conventional CF treatments target the symptoms, precision medicines tackle the underlying genetic mutations which cause the condition.
- Precision medicines are so called because they only treat certain mutations. These drugs are part of a wider move towards personalised healthcare.
Which precision medicines treat cystic fibrosis?
Two precision medicines that help to treat the underlying cause of cystic fibrosis have been licensed for use in the UK, while others are currently being tested on people with CF to determine how they work.
Orkambi (ivacaftor/lumacaftor) targets a mutation that around 50% of people with cystic fibrosis in the UK have and has been shown to improve lung health and reduce hospitalisations. Although it has been licensed for use in the UK, the National Institute of Clinical Excellence (NICE) have not recommended it for use on the NHS, and it is currently only available rarely on compassionate grounds.
Kalydeco (ivacaftor) targets a mutation that under 10% of people with CF in the UK have. Following successful campaigning by the Trust, Kalydeco is available to everyone over the age of two who is eligible and the drug has been shown to significantly increase lung function and reduce hospital admissions and the progression of lung disease .
Other precision medicines
Drug company Vertex have recently released promising results from studies into two drugs, a double combination therapy called Symdeko, and a triple combination therapy.
The double combination therapy (Symdeko) uses a combination of ivacaftor and tezacaftor. The drug has been tested on people with CF with two copies of the F508del mutation and those with one copy of F508del and another mutation, and results have been positive.
The triple combination therapy uses a combination of ivacaftor, tezacaftor and one of three new compounds, and could work in people with one copy of the F508del mutation and one copy of a 'minimal function mutation' - approximately 90% of the CF population.
How are we campaigning for access to these drugs?
The Trust has been campaigning hard to ensure that people with CF can access innovative precision medicines. Find out more about the work we're doing to extend access to precision medicines by clicking on the different drugs below, or join a campaign or explore our campaign timeline and latest news.