Hundreds of children and adults with rare forms of cystic fibrosis in England will be able to access “life-changing” new ‘triple-combination’ therapy for the very first time, the NHS has announced.
Vanzacaftor–tezacaftor–deutivacaftor (Alyftrek®), is the latest in a new generation of therapies to be made available on the NHS that help treat the underlying cause of cystic fibrosis, transforming life expectancy and quality of life for patients.
Following approval by the National Institute for Health and Care Excellence (NICE), patients with the most common form of cystic fibrosis – caused by the F508del mutation – will from today be able to access Alyftrek as an alternative option to existing treatment Kaftrio®.
In a landmark step, NHS England today announced the treatment would also be made available for children and adults with rare forms of cystic fibrosis (who have not previously been eligible for modulator therapy), enabling them to access the latest generation of treatments including Alyftrek and Kaftrio for the first time. This follows NHS England securing a commercial deal with manufacturer Vertex for expanded access beyond the patient group that NICE were able to consider the treatment for.
Cystic fibrosis is a genetic condition that causes thick mucus to build up in the lungs and digestive system, leading to breathing difficulties and serious infections – often requiring intensive treatment and regular hospital admissions.
New ‘triple-combination’ therapy Alyftrek is taken daily at home, and works by correcting the faulty protein that causes the disease to reduce symptoms – helping people live more independently and reducing the time they need to spend in hospital. Alyftrek is a once-daily pill, offering greater convenience for some patients.
Clinical trials have shown Alyftrek to be at least as effective as Kaftrio at improving lung function for people aged 12 years and over with cystic fibrosis caused by a F508del mutation.
While the rare nature of other mutations means there is no clinical data on the effectiveness of Alyftrek, the new NHS England policy will enable doctors to offer access where there is significant unmet clinical need, in line with the approach taken by the European Medicines Agency.
For patients already accessing Kaftrio, the Cystic Fibrosis Medical Association, which represents specialist NHS clinicians, said any decisions to switch to Alyftrek would be made on a case-by-case basis between the person living with cystic fibrosis and their clinical team.
John Stewart, NHS England’s Director for Specialised Commissioning, said: “This is a major leap forward for hundreds of patients living with the rarest forms of cystic fibrosis, offering fresh hope of a better quality of life.
“Access to a once-daily treatment at home can make an enormous difference to patients and their families – reducing the burden of hospital appointments and allowing children and young people to live more freely and independently.
“For those living with the rarest forms of the condition, this represents the very first time they will be able to access this new standard of care that has been so transformative for many since 2019.
“The roll-out of this life-changing therapy demonstrates how the NHS continues to embrace innovation to deliver significant improvements in care for patients across the country at a fair price for the taxpayer”.
Catherine, whose 11-year-old daughter Kate has cystic fibrosis and is currently receiving Kaftrio, said: “We certainly would take the opportunity to make things even better if we can. We are hoping Alyftrek will reduce the need for oral antibiotics and even better still, IVs. An added bonus is that it is a once a day dose.
“People don’t realise what a burden cystic fibrosis is, modulators make things a lot better if you are lucky enough to be able to have them and tolerate them, but CF is still always there”.
NHS England’s latest innovative medicines deal comes days after the publication of the 10 Year Health Plan, with access to cutting-edge treatments for patients helping shift care out of hospital and into the community.
The roll-out means around 95% of people with cystic fibrosis in England are now eligible for modulator therapy.
Patients taking CFTR modulator therapies require regular monitoring including liver function tests, as well as support from multidisciplinary teams to manage their condition and prevent complications.
David Ramsden, Cystic Fibrosis Trust Chief Executive, said: “Today’s announcements are another positive step in the journey to better treatments for everyone with cystic fibrosis – a lifelong, life-limiting condition without a cure. It’s thanks to the incredible support and many years of campaigning of the CF community, and the work of all our partners, that modulator drugs are now a treatment option for thousands of people.
“Today is an important day, but sadly we know that cystic fibrosis continues to make lives too tough and too short, which is why we will continue funding vital research to work towards a future where everyone can benefit from a life unlimited by CF”.
Helen Knight, Director of Medicines Evaluation at NICE, said: “CFTR modulators are already revolutionising the way cystic fibrosis is treated so we’re pleased to be able to recommend Alyfrtek, the latest of this type of treatment that has been shown to be effective, with significant benefits for people with the condition.
“Today’s decision follows NICE’s approval last year of Kaftrio and comes just weeks after Alyfrtek was licensed for use in the UK. This is great news for people with cystic fibrosis and underlies our commitment to getting the best care to patients fast while ensuring the best value for the NHS drugs budget”.
Ludovic Fenaux, Senior Vice President, Vertex International said: “We’re proud that Alyftrek®, our fifth CF medicine, is available today as another treatment option for all eligible CF patients in England. It represents a significant milestone in our journey to serially innovate and further improve the lives of people living with this disease.
“We’re pleased to have reached this agreement with NHS England that recognises the value that this new medicine brings to CF patients, their families and society”.
The NHS is also continuing to support those who are not eligible or cannot tolerate modulator treatments — a group who may need more intensive clinical, psychological, and social care.
Guidance on the expanded range of rare cystic fibrosis gene types that are eligible for Alyftrek is set out in NHS England’s urgent commissioning statement.