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 be able to access Alyftrek as an alternative option to existing treatment Kaftrio.
In a landmark step, NHS England 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.
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.”
