A girl who received a life-changing cystic fibrosis drug has told how she felt better within hours.
Kate Farrer, seven, is one of the first young children to be given Kaftrio on the NHS. Previously, the drug was only available to patients over the age of 12.
However, it has now been extended to the younger age group thanks to a landmark deal completed by the NHS in June 2020.
Cystic fibrosis is a progressive, genetic disease that causes persistent lung infections and limits people’s ability to breathe over time.
But this “modulator” drug benefits lung function, allowing patients to breathe easily and have a better quality of life.
Kate started taking the drug on Sunday, and began to feel an improvement just three hours later, her family said.
She is one of about 1,300 children aged six to 11 who will now be able to access the drug.
Kate said she was “really happy” with the new drug.
“I am really happy because it is a life-changing drug and it is going to help me a lot when I am older.”
Read more:
Mum of young girl with cystic fibrosis speaks of joy at lifesaving drug news
She added that she was feeling “a lot better” and can exhale without coughing.
“Now with this new medication – I have only been on it a few days but I am feeling a lot better now – it is really exciting that I got it, because now life will hopefully be a bit fairer with me, because sometimes it is not as fair because of all of my treatment and medications.
“I am actually quite happy as I can now exhale all the way and not cough at all, not one bit.”
Kaftrio for cystic fibrosis - what we know
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.
Kaftrio also fights off some of the symptoms which affect daily life.
In January, Kaftrio was extended for licence by the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) in England.
It means children aged six to 11 are newly eligible for this cystic fibrosis treatment.
Until this point, Kaftrio was only licensed for those aged 12 and above.
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.”
There are around 8,000 people living with cystic fibrosis in England, which is the second highest proportion in the world.
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