What are we doing to get life-saving drugs?
We have been working alongside the CF community since the first CFTR modulator was licensed in 2015 to get access to all life-saving drugs for the people who need them. Since then, the campaign has intensified, and despite resounding proof as to the effectiveness of these drugs, we have faced huge challenges. After a sustained fight by the CF community and the Trust, Orkambi and Symkevi were finally made available on the NHS at the end of 2019.
We are now working to ensure that everyone with CF in the UK who is eligible for Kaftrio will be able to get access once it receives its European licence.
Find out what’s happened so far in our fight for access to life-saving drugs, and take a look at this list of parliamentarians who have been supportive of our campaign.
What do these drugs do?
Life-saving drugs, also known as precision medicines, CFTR modulators and modulator therapies, work to tackle the underlying cause of cystic fibrosis.
Cystic fibrosis is caused by a faulty gene that affects the production of a protein called CFTR. Its production is affected by different mutations. There are over 2,000 known mutations that can cause CF, and the treatments you can take depend on the mutations you have. Find out more about the science behind what causes cystic fibrosis.
While conventional CF treatments target the symptoms of cystic fibrosis, for example antibiotics that fight infections or steroids that reduce inflammation in the airways, these drugs tackle the underlying genetic mutations that cause CF by helping to make the CFTR protein work effectively. Find out how this happens.
What drugs are currently available?
Three drugs, Orkambi, Symkevi and Kalydeco, are currently available on the NHS after a long fight by the Trust and the CF community, while the latest drug, the triple combination therapy, Kaftrio (known as Trikafta in the US) is currently being assessed by the European Medicines Association (EMA) and deals have been struck in England, Wales, Northern Ireland and Scotland so that it is available on the NHS on the first day of its license which is expected by the Autumn of 2020. Find out what we’re doing to get access to the triple combination therapy, or find out more about some of the life-saving drugs that are available or in the pipeline below.
We recognise that even with access to four CFTR modulator drugs, there will still be people with cystic fibrosis who are not eligible and we will not stop until everyone with cystic fibrosis has access to the most effective medicines. This is why we’re investing in a variety of research projects.
Kaftrio, Vertex's triple combination therapy
The triple combination therapy, Kaftrio (brand name Trikafta in the United States) uses a combination of ivacaftor, tezacaftor and elexacaftor, and targets mutations that approximately 90% of the CF population have. Find out more about the drug and its availability here.
Orkambi (ivacaftor/lumacaftor) targets a mutation that around 50% of people with cystic fibrosis in the UK have. Find out more about the drug and its availability here.
Symkevi (a combination of ivacaftor and tezacaftor) targets multiple mutation combinations that approximately 55% of the UK CF population have. Find out more about the drug and its availability here.
Kalydeco (ivacaftor) targets mutations that under 10% of people with CF in the UK have. Find out more about the drug and its availability here.
Take a look at some frequently asked questions about our fight for access to life-saving drugs, with detailed information on all the different treatments.