One of the first children to receive the game-changing cystic fibrosis treatment, Kaftrio, on the NHS has told how she felt better within hours.
Seven-year-old Kate Farrer started taking revolutionary drug on Sunday, and her family say she started to feel an improvement just three hours later.
The little girl from London is one of about 1300 in the six to 11 age group who are now able to access Kaftrio after the NHS received the green light to roll out the life-changing drug to younger age groups.
For 700 of these children, including Kate, it is the first time they have had access to any of the new cutting-edge generation of cystic fibrosis (CF) drugs, known as modulators'.
Previously, the transformative treatment has only been available to those aged 12 and over, but thanks to a landmark deal secured by NHS England in June 2020, patients automatically benefit whenever the MHRA extends Kaftrio's licence.
Clinicians have now started prescribing the drug to eligible children after the MHRA extended the licence for it, and after stocks of lower-dose Kaftrio specifically for children became available.
Until recently there were no drugs available to treat the underlying causes of cystic fibrosis. Sufferers have now been given the hope of living healthier and longer lives thanks to NHS England's deals for a range of new cystic fibrosis medicines, including Kaftrio which benefit around 90% of CF patients.
Kaftrio significantly improves lung function, helping people with cystic fibrosis to breathe more easily and enhancing their overall quality of life.
Kate, who wants to become an Olympic gymnast, hopes taking Kaftrio will mean not as many tummy aches, fewer trips to hospital, and being able to go on more school trips.
Kate said: I am really happy because it is a life-changing drug and it is going to help me a lot when I am older.
Cystic fibrosis, it means I can sometimes miss out on stuff because I have to go to hospital quite a lot, and the other week I had to miss out on a school trip because it was about rocks and soil and I can't go near soil as it has bacteria in it.
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.
Kate's mum Catherine said: Kate is very sporty, she loves gymnastics, but it has been getting to the point where she can't do the same things as her peers and she is starting to struggle - the past 18 months she has not been in brilliant health with her chest, and she hasn't been able to keep up with people.
Kaftrio has just been amazing - she did a trampoline class and for the first time ever I couldn't hear Kate coughing or clearing her throat. It's just wonderful, it is a real privilege to be able to access this drug and I am just so grateful.
Our hope now with this treatment is that Kate will have more time to live and be a normal seven-year-old. It is a massive relief, CF is progressive and we are needing more treatment, more antibiotics and hospital admissions, and it is starting to impact her more as she gets olderto have it now with the hope that this will hopefully stop CF in its tracks or at least slow it down, her future is really bright.
The CF community is huge and brilliant, all they want is for their children to live a normal life - the CF Trust, the community, the clinicians, everyone has fought for so long to get these drugs and I am just delighted to get to this point, and I can't wait to see more kids get access to it.
Innovative treatments like Kaftrio are life-changing for patients and their families. The latest development will ensure hundreds of children will now be able to access this incredible treatment for the first time.
NHS medical director Professor Stephen Powis said: Thousands of patients are already benefitting from this amazing cystic fibrosis treatment on the NHS, and it is fantastic to see that many more young patients like Kate will now have access to Kaftrio, which can significantly improve lung function meaning activities like running, bike riding, school trips and holidays, and even playing sport with their friends can become easier, greatly improving their quality of life.
The NHS has a strong track record of securing rapid access to innovative, life-changing treatments for our patients and their families, supporting our frontline staff to continue delivering world-class patient care.
Cystic fibrosis is a progressive, genetic disease that causes persistent lung infections and limits the 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.
Dr Cara Bossley, Paediatric Respiratory Consultant at King's College Hospital, said: We're delighted to be able to offer this life-changing treatment to our young patients with cystic fibrosis.
From experience of treating older patients with the therapy, we know it can significantly improve lung function and enhance quality of life.
Nine out of 10 patients with cystic fibrosis - more than 7,000 people in England - can benefit from the new cystic fibrosis therapies, such as Kaftrio, that tackles the underlying causes of the disease as well as symptoms.
David Ramsden, Chief Executive at the Cystic Fibrosis Trust, said: It's fantastic that children aged six to 11 right across the UK are now starting to receive Kaftrio. It is important that the roll out moves quickly so that all children who are eligible can start to benefit from this life changing medicine.
We also recognise that these drugs won't work for everyone, and Cystic Fibrosis Trust will continue working to find new treatments that help every child.
Described by patient groups as a revolutionary drug', Kaftrio 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.
The Kaftrio deal is one of a series of medicine deals made possible by the NHS' advanced commercial capabilities, which have also secured patient access to cutting-edge one-shot gene therapies like life-saving drug Zolgensma, which can help babies with Spinal Muscular Atrophy move and walk; and the first new treatment for sickle cell disease in more than two decades.