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Fighting for life-saving drugs

We are working with the cystic fibrosis community to make life-saving drugs available on the NHS to the people who need them. Find out how you can help.

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?

Three 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

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

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.

Vertex's triple combination therapy

The triple combination therapy (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.

Kalydeco

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.

How are we campaigning for access to these drugs?

The Trust has been campaigning hard to ensure that people with CF can access all CFTR modulators. Find out more about the work we're doing to extend access to precision medicines, find out how we're campaigning in your areajoin a campaign or explore our campaign timeline and latest news.

Supportive parliamentarians

Take a look at the list of parliamentarians who have supported our campaign for access to life-saving drugs.

Orkambi

Orkambi is the second precision medicine to be licensed in the UK for people with CF and treats the F508del mutation, which around 50% of people with CF have. It is not currently available on the NHS.

Kalydeco

Kalydeco is the first precision medicine to be available on the NHS and is effective in just under 10% of people with cystic fibrosis. Kalydeco is currently available to every eligible person over the age of two in the UK.

Other precision medicines

A double combination therapy and a triple combination therapy are currently being trialed to see how they could help to treat up to 90% of people with cystic fibrosis. Recent results are positive.