The NHS will provide a new triple combination therapy to hundreds of children and adults with cystic fibrosis in England, aiming to improve lung function and quality of life for eligible patients
Hundreds of children and adults living with cystic fibrosis in England are set to benefit from a newly approved triple combination therapy, following an announcement by NHS England. The rollout marks a significant advancement in treatment, offering improved health outcomes and greater quality of life for eligible patients.
Transforming life expectancy
Vanzacaftor–tezacaftor–deutivacaftor (Alyftrek®) is a new generation therapy that helps treat the underlying cause of cystic fibrosis, improving quality of life for patients.
Patients with the most common form of cystic fibrosis, which is caused by the F508del mutation, will be able to access Alyftrek from today, as an alternative to the existing treatment Kaftrio.
The treatment will 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 was able to consider the treatment for.
Correcting the faulty protein that causes cystic fibrosis
The therapy is administered daily at home and works by correcting the faulty protein that causes the disease, thereby reducing symptoms and helping people live independently.
Clinical trials, which involved a large number of participants and were rigorously conducted, 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. This approach is in line with the standards set by the European Medicines Agency, a reputable regulatory body for medicines in the European Union.
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 rollout 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”.
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”.
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”.
