Hundreds of people with rare forms of cystic fibrosis will gain access to a new once-daily therapy through the NHS for the first time.
Cystic fibrosis is a genetic condition that causes thick mucus to build up in the lungs and digestive system, leading to infections, breathing problems and digestive issues.
The new treatment, a triple-combination therapy called vanzacaftor–tezacaftor–deutivacaftor (branded Alyftrek), targets the faulty protein that causes the condition.
Following approval by the National Institute for Health and Care Excellence (NICE), patients with the most common cystic fibrosis mutation, F508del, will from today be able to access Alyftrek as an alternative to the current treatment Kaftrio.
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.”
NHS England has also announced a commercial deal with manufacturer Vertex to expand access to both Alyftrek and Kaftrio for children and adults with rare forms of cystic fibrosis who were previously ineligible for CFTR modulator therapies – drugs that correct the defective protein function.
The once-daily pill is taken at home and offers convenience alongside clinical benefits.
It aims to improve lung function and reduce symptoms, helping patients live more independently and spend less time in hospital.
Clinical trial data showed Alyftrek to be at least as effective as Kaftrio for people aged 12 and over with the F508del mutation.
While there is limited trial data for rarer mutations, the new NHS England policy allows clinicians to offer access in cases of significant unmet clinical need, in line with the approach of the European Medicines Agency.
The rollout comes just days after the publication of the NHS’s 10-Year Health Plan, which includes shifting more care into the community and increasing access to advanced treatments.
The Cystic Fibrosis Medical Association said decisions about switching from Kaftrio to Alyftrek would be made case by case between patients and clinical teams.
David Ramsden, chief executive of the Cystic Fibrosis Trust, 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.”
