NHS Expands Access to Once-Daily Cystic Fibrosis Treatment - EMJ

NHS Expands Access to Once-Daily Cystic Fibrosis Treatment

THE NHS has announced the nationwide roll-out of Alyftrek® (vanzacaftor–tezacaftor–deutivacaftor), a new once-daily ‘triple-combination’ therapy for cystic fibrosis, extending access to hundreds of patients, including those with rare genetic mutations previously ineligible for similar treatments.

Approved by the National Institute for Health and Care Excellence (NICE), Alyftrek is designed to target the underlying cause of cystic fibrosis, a faulty protein that leads to thick, sticky mucus in the lungs and digestive tract. It has shown comparable efficacy to Kaftrio®, the current standard therapy for patients with the common F508del mutation, in improving lung function in clinical trials involving individuals aged 12 years and over.

In a significant expansion of care, NHS England will also offer Alyftrek® and other modulator therapies to patients with rare forms of cystic fibrosis. Although clinical trial data are lacking for these rarer mutations, doctors can now prescribe the treatment where there is a high unmet clinical need, aligning with European Medicines Agency (EMA) guidance.

Alyftrek® is taken once daily at home, offering a more convenient option for some patients. For individuals and families managing the daily burden of cystic fibrosis, reduced hospital visits and improved independence could be transformative. However, switching from Kaftrio® to Alyftrek® will be assessed on a case-by-case basis by clinical teams.

With this roll-out, approximately 95% of people with cystic fibrosis in England are now eligible for cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapy. These patients require ongoing monitoring, including liver function tests and support from multidisciplinary teams.

Patient advocacy groups welcomed the announcement. David Ramsden, Chief Executive of the Cystic Fibrosis Trust, called it “another positive step” in expanding access to vital treatments, while noting the ongoing challenges faced by those still without options.

NHS leaders emphasised that this development reflects broader goals outlined in the 10-Year Health Plan, focusing on innovative, community-based care.

While today’s announcement marks a meaningful step forward, the NHS continues to invest in research and support for individuals who are not yet eligible or cannot tolerate modulator therapies, ensuring that all people living with cystic fibrosis are supported on their treatment journey.

Reference

NHS England. NHS to roll-out new ‘triple combination’ therapy for hundreds of children and adults with cystic fibrosis. 2025. Available at: https://www.england.nhs.uk/2025/07/nhs-to-roll-out-new-triple-combination-therapy-for-hundreds-of-children-and-adults-with-cystic-fibrosis/. Last accessed: 18 July 2025.

 

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