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Green light for Kalydeco

NHS England gives the go-ahead for transformational treatment that can halt the progression of cystic fibrosis for two-to-five-year-olds.

After a sustained campaign by the Cystic Fibrosis Trust, young children in England will receive Ivacaftor (Kalydeco), a life-changing drug for cystic fibrosis (CF). Kalydeco is proven to significantly improve lung function and slow the progression of CF, reducing time spent on intravenous antibiotics from five weeks to less than five days a year. It has also been found that the earlier Kalydeco is provided, the better the outcome for individuals. Data also suggests for the first time that people receiving this treatment could have a near normal life expectancy.

Kalydeco has been provided in England for people over the age of six since 2014. It was licensed for two-to-five-year-olds in November, and today NHS England have announced that it will be one of the drugs offered on the NHS to eligible two-to-five-year-olds with CF in England following the ‘re-prioritisation process’. Kalydeco is already being offered to children in Scotland, and it is expected that Northern Ireland and Wales will now follow suit.

The first precision medicine in CF, Kalydeco targets a group of rare mutations that affect about 5% of people with the condition in the UK. Emma Foord, from Wiltshire in Southwick, has a 22-month-old son, William, who could benefit from the drug:

“I’m over the moon to hear that William will be given access to Kalydeco - this drug could have a huge impact on my son’s future. I have watched him fight for his life on three occasions as a result of cystic fibrosis. I have nearly lost him to a perforated bowel, a bowel obstruction and septic shock as a result of a ruptured bowel abscess. I have also seen him go to an operating theatre on four occasions, two of which were lifesaving, so to know that he will be given access to this life-changing drug is a huge relief. My biggest worry is CF winning and my son losing. This gives me great hope that it will help normalise William’s life now and in the future.”

James Barrow, Head of External Affairs at the Trust, said:

“After a year of waiting we are pleased that this life-changing treatment is now available to young children in England. It will transform lives and give hope for the future. The drug is also available in Scotland and we urge Wales and Northern Ireland to follow suit."

The Trust is campaigning hard to ensure innovative drugs like Kalydeco are provided to those who need them. We are funding vital work into clinical trials to help unblock the pipeline of exciting new treatments that could help improve the lives of people with cystic fibrosis.

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