What is Kalydeco?
Kalydeco is the first precision medicine for cystic fibrosis to become available on the NHS and is also known by the name ivacaftor.
Who does Kalydeco work on?
Kalydeco is effective in anyone with at least one gating mutation, such as G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P or G139D. This accounts for roughly 5% of the population of people with cystic fibrosis in the UK. Kalydeco helps to open ‘gates’ in the cells of people with these mutations, allowing chloride to move into and out of the cells and helping to keep the balance of salt and water in the lungs.
Find out more about mutations with our useful video.
What effect does Kalydeco have?
Outcomes calculated using data from the UK Cystic Fibrosis Registry show that median FEV1% (forced expiratory volume – a measure of lung function) increased from an average of 55.4 to an average of 64.1; a significant improvement. People with CF often have higher levels of chloride in their sweat; tests on 439 people showed that their sweat chloride levels dropped by almost 50% on average after taking Kalydeco.
Who is currently receiving Kalydeco in the UK?
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. If you are currently receiving Kalydeco there is no risk that this treatment will be withdrawn as a result of further negotiations.
What are we doing about it?
Stay up to date with Stopping the Clock, our dedicated campaign to put pressure on the Government, NHS and pharmaceutical companies to ensure these drugs reach the people who need them without delay, join the campaign and get involved yourself by taking part in campaigning in your area.