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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 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. Six weeks later NHS England announced that people with CF in England will receive access to Orkambi and Symkevi as part of a two-year managed access agreement with Vertex, and shortly afterwards the Department of Health in Northern Ireland has confirmed its intention to complete a similar deal, the plans for which are now being finalised. On 13 November, the Welsh Government announced that a deal has been agreed in principle for people with CF living in Wales.

Kalydeco

Kalydeco boxes

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 for everyone over the age of 12 months with one of nine rare gating mutations (G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P or G139D), and to people aged 18 years and older who have the R117H mutation. The drug has been shown to significantly increase lung function and reduce hospital admissions and the progression of lung disease.

Symkevi

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. Six weeks later NHS England announced that people with CF in England will receive access to Orkambi and Symkevi as part of a two-year managed access agreement with Vertex, and shortly afterwards the Department of Health in Northern Ireland has confirmed its intention to complete a similar deal, the plans for which are now being finalised. On 13 November, the Welsh Government announced that a deal has been agreed in principle for people with CF living in Wales.

Trikafta

The triple combination therapy (brand name Trikafta in the United States) uses a combination of ivacaftor, tezacaftor and elexacaftor, 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. Recently, Trikafta was approved in the United States for those aged 12 and over who have at least one F508del mutation. Vertex have submitted Trikafta to the EMA, who are analysing it for safety and clinical effectiveness, with further updates expected in 2020.

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, 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.