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Scotland leads the way with Kalydeco for younger children

The first UK country grants access to life-changing treatment to two-to-five-year-olds.

In Scotland, children aged two to five with G551D or one of nine similar cystic fibrosis (CF) mutations are now able to access Kalydeco, in addition to older children and adults who are already receiving the drug. 

Kalydeco, the first precision medicine for CF, offers significant health benefits to around 5% of the UK CF population who have some forms of the mutation that causes the condition. It is proven to significantly improve lung function and slow the progression of CF, reducing time spent in bed on intravenous antibiotics from five weeks to less than five days a year on average. 

Kalydeco has been provided in Scotland since 2014 for people over the age of five. It was licensed for two-to-five-year-olds in 2015 and is now being provided to those children in Scotland who require it.

England, Northern Ireland and Wales are yet to follow suit and offer this treatment to those who need it. NHS England did recommend that Kalydeco should be made available to two-to-five-year-olds in England, but the final decision was put on hold following the outcome of the judicial review regarding the antiviral drug for the prevention of HIV known as PrEP.

Doreen MacEachen, from Glasgow, mother to three-year-old Hugo who has been taking Kalydeco for around six weeks, said: “We are extremely grateful for this drug. Since he’s been on it we’ve noticed improvements in his health - he seems to have more energy, he recently had a cold which he recovered from easily, his stools look more normal and I'm sure he has put on weight – it’s fantastic!”

Emma Foord’s 21-month-old son William is from England and could benefit from the drug. She said: “It’s devastating that William isn’t being given access to Kalydeco - this drug could have a huge impact on my son’s health at such a crucial stage in his development. I have watched him fight for his life on three occasions. I want him to be able to achieve and conquer whatever his heart desires, without cystic fibrosis holding him back.” 

James Barrow, Head of External Affairs at Cystic Fibrosis Trust said: “We are pleased that this life-changing drug is now available to children in Scotland but disappointed it is not available in other parts of the UK. It is inconceivable that a drug that has been shown to slow the progression of cystic fibrosis would be denied for younger people who stand to benefit so much at such a crucial time in their development. The situation in England is particularly cruel as NHS England said the treatment should be available but then put the decision on hold. We urge England, Wales and Northern Ireland to follow Scotland’s lead."

Our Stopping the Clock campaign calls for access to precision medicines for everyone who could benefit from them, wherever they live in the UK. Add your voice to the campaign now.