‘Life-changing’ Cystic Fibrosis treatment trialled in Manchester now approved for NHS use

A new once-daily cystic fibrosis (CF) treatment trialled in Manchester has been approved for NHS use, following a recommendation made earlier this month by the National Institute for Health and Care Excellence (NICE).

Clinical trials delivered at the National Institute for Health and Care Research (NIHR) Manchester Clinical Research Facility (CRF) have shown the new triple therapy to be at least as effective as current drug Kaftrio® at improving lung function for people aged over 12 with CF caused by a specific gene (F508del) mutation.

The NIHR Manchester CRF at Wythenshawe Hospital, was the top-recruiting UK site for the adult study for the modulator therapy Vanzacaftor–tezacaftor–deutivacaftor, also known as Alyftrek®, which helps target the underlying cause of the disease.

Professor Alex Horsley, Medical Director of the NIHR Manchester CRF at Wythenshawe Hospital, was the Lead Investigator. He said: “What we have seen in this study is that this new triple combination therapy is as good for lung function. We have also seen a slightly bigger impact on sweat chloride levels, which means it likely has a better effect upon the core problem in CF.

“As the treatment is taken just once daily, this could also make a big difference to people with CF, with more convenience and flexibility, helping simplify their treatment regimens, which are already very complicated.”

Cystic Fibrosis is a rare genetic condition affecting more than 10,000 people in the UK. It causes thick, sticky mucus to build up in the lungs, digestive system and other organs causing a wide range of challenging symptoms. The condition is caused by reduced quantity and/or function of the CF transmembrane conductance regulator (CFTR) protein that regulates levels of sodium and chloride in cells. A person will be born with CF when the faulty CFTR gene from both parents is genetically passed on.

Adam’s story

Adam Higson, 45, from Rochdale was diagnosed with CF in 1980, when he was 11-months old. He took part in the clinical trial at the NIHR Manchester CRF at Wythenshawe Hospital. 

He said: “I still remember driving home after taking my first tablet at hospital and I could already feel in my lungs it was working. I feel full of energy and when I run now it feels like I’ve got a jet pack on my back. 

“The main impact of CF on my life was a persistent cough, which was particularly annoying at night and would disturb my sleep. Despite that I always kept myself fit and healthy by taking part in sports and doing exercise.”

Adam, who is a keen runner and works in Communications, added: “My experience taking part in clinical trials has been incredible and life changing. The specialists at the hospital are global experts with incredible knowledge.”

Adam, who has completed six marathons with more planned in 2025, says the therapy has enabled him to improve his personal best running times.

He said: “The once daily therapy is much, much more convenient. The tablet is only taken in the morning, and you must have food with it. When it was twice a day it
was much harder to comply if my plans changed.”

The trial examined whether the new drug combination, administered once daily as a tablet, worked as well as Kaftrio. The study took place over a year with around 1,000 patients recruited globally at more than 100 different sites.

Results were published earlier this year in the Lancet Respiratory Medicine Journal. In March, the Medicines and Healthcare products Regulatory Agency (MHRA) approved the triple combination medicine to treat CF in people aged six years and older who have specific CFTR gene mutations.

Professor Horsley, respiratory consultant at Wythenshawe Hospital, part of Manchester University NHS Foundation Trust, and Investigator with the NIHR Manchester Biomedical Research Centre (BRC) Respiratory Theme, added: “We also hope that better correction of the underlying deficit in CFTR function may lead to better long-term impact for patients.

“The improved correction means that it also now works for some CF gene changes that Kaftrio was not effective against, meaning that more people can potentially benefit from this treatment.”

Earlier this month, NHS England announced the treatment would also be made available for children and adults with rare forms of CF, who had not previously been eligible for modulator therapy.

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.”