‘Miracle’ cystic fibrosis treatment for children on the NHS

The NHS has announced that hundreds of children are set to benefit from a ‘miracle’ treatment for cystic fibrosis, which has already changed the lives of thousands of people.

Patients in the UK were some of the first in Europe to benefit from Kaftrio when NHS England secured a landmark deal in June 2020. Nine out of 10 patients with cystic fibrosis – more than 7,000 people in England – can benefit from the ‘triple therapy’, which tackles the underlying causes of the disease as well as symptoms.

Kaftrio significantly improves lung function, helping people with cystic fibrosis to breathe more easily and enhancing their overall quality of life.

Now, the MHRA confirmed an extension to the licence for Kaftrio, which means more than 1,300 children in England with cystic fibrosis, aged six to 11, are newly eligible for this treatment. For almost 700 of these children it will be the first time they are able to receive one of a new generation of cystic fibrosis drugs, known as ‘modulators’.

Amanda Pritchard, NHS England chief executive, said: “Since NHS staff delivered one of the fastest rollouts of Kaftrio in the world just over a year ago, the lives of thousands of patients with cystic fibrosis have been transformed. Innovative treatments like Kaftrio are life-changing for patients and their families, and that is why the NHS has done all it can since we secured the deal for Kaftrio to ensure patients benefit as soon as possible.

“The latest development will ensure hundreds of children will now be able to access this incredible treatment for the first time. The NHS Long Term Plan committed to using the latest cutting-edge treatments and therapies to save and improve patients’ lives, and this is the latest example of NHS England using the advanced commercial capabilities we have been given to deliver this.”