People in England will be among the first in Europe to access a new once-daily treatment for cystic fibrosis, following a recommendation in final draft guidance from the National Institute for Health and Care Excellence (NICE).
The triple combination medicine vanzacaftor/tezacaftor/deutivacaftor (brand name Alyftrek), which is manufactured by Vertex Pharmaceuticals, was licensed for use in the UK in March 2025 and will now become available immediately through the NHS in England.
It will be an option for people aged six years and over with at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
The recommendation comes after a rapid assessment by NICE that compared Alyftrek with elexacaftor/ivacaftor/tezacaftor (brand name Kaftrio) – Vertex’s other NICE-approved triple therapy.
The rapid assessment concluded that Alyftrek is at least as effective as Kaftrio and available at a similar cost, but offers the additional benefit of a once-daily regimen compared to Kaftrio’s twice-daily dosing.
In 2023, the license of Kaftrio was extended by the Medicines and Healthcare products Regulatory Agency to include children aged two to five years old.
Accessing a new standard of care for CF
Vanzacaftor and tezacaftor act as CFTR correctors, increasing the amount of CFTR protein on the cell surface, while deutivacaftor improves the activity of the protein at the cell surface.
Together, these actions reduce the thickness of lung mucus and digestive fluids, helping to treat the underlying cause of cystic fibrosis and relieve symptoms.
Commenting on the NICE approval, 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.’
Helen Knight, director of medicines evaluation at NICE, said the decision was ‘great news for people with cystic fibrosis’ and showed commitment to ‘getting the best care to patients fast while ensuring the best value for the NHS drugs budget’.
Ms Knight continued: ‘CFTR modulators are already revolutionising the way cystic fibrosis is treated so we’re pleased to be able to recommend Alyftrek, the latest of this type of treatment that has been shown to be effective, with significant benefits for people with the condition’.
David Ramsden, chief executive of the Cystic Fibrosis Trust, noted this ‘important’ development but added: ‘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.’
Last year, a phase 3 open-label study found ivacaftor (brand name Kalydeco) is safe and effective in infants aged four weeks and over, paving the way for earlier initiation of therapy for newborns diagnosed with the condition.
A version of this article was originally published by our sister publication The Pharmacist.
