Breaking news: On 12 September 2019, the Scottish Government reached an agreement with Vertex with support from the UK CF Registry and announced a five-year deal that will see access to Orkambi and Symkevi for everyone with cystic fibrosis in Scotland who stand to benefit from the drugs.
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. Orkambi is licensed for use in the UK, and in September 2019 the Scottish Government announced a five-year deal giving access to Orkambi and Symkevi for everyone with CF in Scotland who can benefit from them. However, the drugs have still not been recommended for use on the NHS in England, Wales or Northern Ireland, and are currently only available rarely on compassionate grounds and through limited clinical trial access.
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 .
Symkevi (a combination of ivacaftor and tezacaftor) targets multiple mutation combinations. These include people with two copies of F508del (which around 50% of people with CF in the UK have) and people with F508del and one of 14 less-common 'residual function' mutations (approximately 5% of people with CF in the UK). This treatment has been shown to improve lung function and reduce hospitalisations.
Symkevi was licensed for use by the EMA in November 2018, and in September 2019 the Scottish Government announced a five-year deal giving access to Symkevi and Orkambi for everyone with CF in Scotland who can benefit from them. However, the drugs have still not been recommended for use on the NHS in England, Wales or Northern Ireland, and are currently only available rarely on compassionate grounds and through limited clinical trial access.
Triple combination therapy
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, finding out how we're campaigning in your area, joining a campaign or exploring our campaign timeline and latest news.
Take a look at the list of parliamentarians who have supported our campaign for access to life-saving drugs.