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Cystic Fibrosis Trust

Kalydeco

Kalydeco (also known as ivacaftor) is the first precision medicine for cystic fibrosis to become available on the NHS.

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?

Kalydeco is available to everyone over the age of two who is eligible. If you are currently receiving Kalydeco there is no risk that this treatment will be withdrawn as a result of further negotiations.

Why is Kalydeco not available to everyone who needs it?

The processes involved in making drugs available to the people they could help are complicated. Creating and testing a drug like Kalydeco is very expensive and a lack of long-term data can make it hard for NICE, the body that appraises these drugs for use in the NHS, to accurately assess their cost-effectiveness in order to make them available. 

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.

For the latest news on our Stopping the Clock campaign and other updates in the CF community, sign up to receive our e-newsletter.

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