‘Miracle’ Cystic Fibrosis Treatment For Children on The NHS
Hundreds of children are set to benefit from a ‘miracle’ treatment for cystic fibrosis, which has already changed the lives of thousands of people, the NHS has announced today.
Patients here 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.
The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has now confirmed an extension to the licence for Kaftrio, which means more than 1300 children in England with cystic fibrosis, aged six to 11, are newly eligible for this treatment.
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.”
Health and Social Care Secretary Sajid Javid said: “I’m delighted that hundreds more children will receive this life-changing cystic fibrosis drug, which can give them a better quality of life for longer.
“Thanks to NHS staff, thousands of people have already benefitted as part of one of the fastest rollouts of Kaftrio in the world – and I salute them on this incredible accomplishment.”
Until now Kaftrio was only licensed for those aged 12 and above.
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’.
June Raine, MHRA chief executive, said: “It’s very good news that after a rigorous review, we have approved Kaftrio for children aged six to 11, enabling the NHS to bring this life-saving treatment to more children suffering from cystic fibrosis.
“We will continue to work closely with our colleagues across the health system to help bring cutting-edge medicines to patients.”
With today’s extension to the licence the company will be able to supply the new dose of Kaftrio to the NHS in England within weeks, enabling clinicians to then prescribe to all young patients aged six to 11 where it is considered the best option for their treatment.
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.
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.
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.
The terms of NHS England’s deal meant that patients would automatically be entitled to access Kaftrio under all future extensions of the drug’s licence.
Latest News from
NHS top doctor rallies remaining people due a spring booster ahead of final COVID jab drive23/06/2022 11:20:00
England’s leading NHS medic is urging those eligible for a spring booster but yet to come forward to take up the offer as soon as possible as a quarter of a million reminders go out before the end of the month.
Medical support worker role helps hundreds of refugees to become NHS doctors17/06/2022 12:25:00
Hundreds more international medics and refugees, including those from Ukraine, Afghanistan and Myanmar have joined the health service and are set to become NHS doctors, thanks to the introduction of a new medical support worker role (MSW).
Thousands of the longest waiters get treatment as NHS drives down long waits for care17/06/2022 09:15:00
The number of people on the waiting list for diagnostic tests has dropped and there are two-thirds fewer people waiting more than two years for elective care, new figures out yesterday show, as the NHS continues to make progress in the biggest catch-up in health service history.
NHS lands breakthrough in global battle against superbugs15/06/2022 16:25:00
The NHS is set to roll out two ‘superbug’ busting drugs through a world-first, pioneering subscription deal that will help tackle antimicrobial resistance, the head of the NHS announced today.
High street pharmacies spot cancers in new NHS early diagnosis drive15/06/2022 15:05:00
Staff in high street pharmacies will be funded to spot signs of cancer as part of a new drive to catch tumours early when they are easier to treat, NHS chief executive Amanda Pritchard announced today.
Health and Care Staff Honoured in NHS Parliamentary Awards14/06/2022 14:05:00
Nurses, midwives, doctors, vaccination teams and domestic staff are among those celebrated in a prestigious competition to mark the NHS’s 74th birthday, as the NHS Parliamentary Awards shortlist is revealed.
GP surgeries to provide specialist mental health support14/06/2022 09:15:00
Thousands of new mental health experts will be on hand to support people in their local GP practice amid record demand for services, the head of the NHS announced recently (11 June 2022).
One million checks delivered by NHS ‘one stop shops’06/06/2022 14:15:00
NHS ‘one stop shops’ have delivered over one million checks and tests since the rollout began, as the biggest catch up programme in health history gathers pace.
Open letter from NHS, charity and community leaders to people with a weakened immune system27/05/2022 13:20:00
We join together as NHS and charity leaders to encourage people with a weakened immune system to continue to book in or visit a walk-in centre for their COVID-19 vaccines. All individuals aged 12 years and over who are immunosuppressed are advised to receive a Spring booster dose of the vaccine, typically six months after their last dose.