A treatment for people who have cystic fibrosis is now available to more than 1,000 children on the NHS.
Kaftrio, which tackles the underlying causes of the disease as well as symptoms, has been extended for licence by the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) in England.
It means more than 1,300 children with cystic fibrosis, aged six to 11, are newly eligible for this treatment.
They will be able to start receiving it within weeks.
David Ramsden, Chief Executive at the Cystic Fibrosis Trust, said: "Today the MHRA and European Commission has agreed to license #Kaftrio for use in children aged 6- 11 years old." (1/3) pic.twitter.com/5fLTZKzd2n
— Cystic Fibrosis Trust (@cftrust) January 11, 2022
Until now Kaftrio was only licensed for those aged 12 and above.
NHS England first secured a deal to offer it to patients in June 2020.
Kaftrio, described by patient groups as a “revolutionary drug”, is a triple combination treatment combining three drugs which perform different functions – ivacaftor, tezacaftor and elexacaftor – and tackles the underlying causes of the disease, by helping the lungs work effectively.
NHS chief executive Amanda Pritchard 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.”
Cystic fibrosis is a progressive, genetic disease that causes persistent lung infections and limits people’s ability to breathe over time.
There are around 8,000 people living with cystic fibrosis in England, which is the second highest proportion in the world.