Delight at wonder drug OK for 1,500 children with CF

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SOME 1,500 children with cystic fibrosis were celebrating yesterday after finally winning access to wonder drug Kaftrio.

The Daily Express has fought since 2019 for the availability of US firm Vertex’s medication, hailed as “almost a cure” for the condition.

In 2020, the NHS unveiled a landmark Kaftrio deal but, until now, only those aged 12 and over could take it.

But yesterday it was finally approved for children across the UK aged six to 11, meaning 1,500 primary school youngsters will soon be on the life-extending drug.

Out of that number, around 400 children will be taking their first CF treatment, having not been able to take Vertex’s earlier drugs Orkambi and Symkevi. For most families, the shock announcement was the culmination of years of fighting and waiting.

Overwhelmed Marta Kiszka, 35, of Canterbury, Kent – mother of Darcie, six – said: “We’ve been waiting for that for the last six years.

“This is like the feeling of being on the edge of something huge. Something game-changing. It will save her life.”

Alison Fare, 40, of Conwy, North Wales – mother to CF-suffering daughters Imogen, nine, and Annabelle, six – said: “When Imogen was born in 2012 there were no CF drugs and at diagnosis I felt her future looked very bleak and scary.

“Fast forward to 2022 and, after already trying Orkambi and Symkevi, they can soon be on the real icing on the cake, Kaftrio.

“Now I look forward to their

future with less fear and dread and more with hope.”

There are around 10,600 Britons with CF, which destroys the lungs with sticky mucus. But 90 per cent can thrive if given the miracle pill.

Desperate families have been waiting for the Medicines and Healthcare products Regulatory Agency to approve it for younger children.

Yesterday, they finally gave it the green light, as did the European Medicines Agency, who approve Northern Ireland’s drugs.

David Ramsden, the chief

executive of the Cystic Fibrosis Trust, said: “This is another hugely significant moment for people with CF.

“This is a victory the CF community fought long and hard for and families will be celebrating today, and I am celebrating with them.”

The Daily Express and the CF Trust have now vowed to fight for the 10 per cent of sufferers with rare genes who cannot take Kaftrio.

Mr Ramsden added: “The Trust won’t stop working, so everyone has access to life-changing treatments.”