What you need to know about Kalydeco
Precision medicines for cystic fibrosis treat the underlying genetic mutation that causes the condition, whereas traditional medicines treat only the symptoms. This means that precision medicines can have a huge impact on quality of life by reducing the amount of time spent in hospital and slowing the decline in lung function experienced by people with cystic fibrosis.
There are many different mutations that can cause cystic fibrosis, and each person with the condition will have two of these. They can be the same, or two different mutations in one individual. Precision medicines are so-called because they treat only certain genetic mutations, rather than a symptom such as pancreatic insufficiency, which could affect everyone with cystic fibrosis. These drugs are part of a wider move towards personalised healthcare.
Kalydeco is the first precision medicine for cystic fibrosis to become available on the NHS and is also known by the name Ivacaftor.
What is Kalydeco?
Kalydeco is a precision medicine that works on gating mutations, which just under 5% of people with CF in the UK have. It 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.
Who does Kalydeco work on?
Kalydeco is effective in anyone with at least one gating mutation, such as G551D, G187R, S549N, S549R, G551S, G1244E, S1251N, S1255P or G139D. This accounts for roughly 5% of the population of people with cystic fibrosis in the UK.
Who is currently receiving Kalydeco in the UK?
- June 2012: Kalydeco is prescribed as a treatment for people over the age of six with at least one copy of the G551D mutation.
- July 2012: Kalydeco is prescribed for people over the age of six with at least one copy of the following gating mutations: G187R, S549N, S549R, G551S, G1244E, S1251N, S1255P or G139D.
- October 2016: Kalydeco is prescribed for two-to-five year olds in Scotland with at least one copy of the above mutations.
- Between July 2012 and December 2015, 439 patients in the UK have been prescribed Kalydeco.
Kalydeco is available to everyone over 6 who is eligible, and we soon expect a decision on children aged 2-5. If you are currently receiving Kalydeco there is no risk that this treatment will be withdrawn as a result of further negotiations.
What does Kalydeco do?
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.
Why isn't it 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 is the Cystic Fibrosis Trust doing?
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.