Hundreds of children and adults with cystic fibrosis can access “life-changing” next gen wonder drug Alyftrek for the first time from today, after NHS England finally agreed a landmark deal. CF is a life-limiting genetic condition that slowly clogs the lungs with thick mucus, with over 11,000 sufferers in the UK – but before 2019 the NHS has no effective treatment.
In 2024 – after a five year Daily Express campaign – US drug giant Vertex agreed an NHS deal so the 90 per cent of CF patients suitable for their treatments – Orkambi, Symkevi and Kaftrio – could access them. But after the National Institute for Health and Care Excellence (NICE) said Vertex’s new once-daily treatment Alyftrek is more effective than twice-daily Kaftrio, NHS England (NHSE) patients with the common CF mutation F508del aged six and older can now access the next gen drug from today.
Alyftrek – also called the ‘vanza triple’ due to the triple-combination of drugs vanzacaftor, tezacaftor and deutivacaftor – has shown startling tests in US studies – with hopes of further boosting life expectancy and quality of life for patients.
The new miracle drug works by correcting the faulty protein that causes the cruel disease in the first place and the deal follows an agreement with Vertex for expanded access that still includes their other CF drugs.
It’s also hoped some of the small percentage of patients unable to access any treatments due to the rarity of their faulty CF genes may be able to take Alyftrek – with NHSE saying: “While the rare nature of other mutations means there is no clinical data on the effectiveness of Alyftrek, the new NHSE policy will enable doctors to offer access where there is significant unmet clinical need.”
The Daily Express first featured young gymnast and then seven year-old Kate Farrer, of East Dulwich, south-east London, back in 2022 when she was thrilled to start taking Kaftrio.
Now excited mum Catherine says Kate, now aged 11, could have an even better quality of life on Alyftrek than she already has on recent wonder pill Kaftrio.
Catherine, 41, explained: “We certainly would take the opportunity to make things even better if we can.
“We are hoping Alyftrek will reduce the need for oral antibiotics and even better still, IVs. An added bonus is that it is a once a day dose.
“People don’t realise what a burden cystic fibrosis is, modulators make things a lot better if you are lucky enough to be able to have them and tolerate them, but CF is still always there.”
For patients already accessing Kaftrio, any decisions to switch to Alyftrek would be made on a case-by-case basis between the patient and their NHSE clinical team.
NHS England’s latest innovative medicines deal comes days after the publication of the 10 Year Health Plan, with access to cutting-edge treatments for patients helping shift care out of hospital and into the community.
NHSE says the roll-out “means around 95 per cent of people with cystic fibrosis in England are now 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.”
In the US, Dr Jordana Hoppe, associate professor of paediatrics-pulmonary medicine at the University of Colorado School of Medicine, led a paediatric study into the safety and efficacy of Alyftrek on 78 children with CF.
At first they were on Kaftrio and then switched to the new, once-daily pill – and most with startling results superior to those from Kaftrio.
Patients experienced an average decrease of eight points in sweat chloride values, the measurement used to diagnose cystic fibrosis based on the restoration of CFTR function.
Dr Hoppe explained: “To say someone has CF, they have to have a sweat chloride test of 60 or higher. The percentage of participants that had a sweat chloride below 60, the diagnostic criteria for CF, and below 30, a sweat chloride value that is in the normal range, is higher than the clinical trials of (Kaftrio).”
CF is a genetic disorder that causes severe damage to the lungs, digestive system and other organs. It’s caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
The CFTR protein produces thick and sticky mucus in various organs, which can block airways and passageways and cause severe complications.
CFTR-modulator drugs, such as Alyftrek, target the defective protein to help improve lung function, lower the risk of respiratory infections, and reduce symptoms.
It is also hoped Alyftrek may reduce the physical and mental side effects that some patients on Kaftrio have suffered – prompting some UK patients coming off Kaftrio or reducing their intake.
David Ramsden, Cystic Fibrosis Trust Chief Executive, told us: “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.”
The Vertex drugs have rocketed the health of sufferers, with some gravely-ill patients in intensive care that the Daily Express urgently highlighted are now not only healthy – but have had babies.
In December 2019, 28 year-old Nicole Adams was fighting for every breath in Belfast City Hospital’s ICU with just 10 percent of her lungs working and her tearful loved-ones preparing to say their final goodbyes.
At one stage she removed her oxygen mask to ring us to say: “I’m so scared – I’m dying. What can I do?” – before weeping and trying to sing Elton John’s hit ‘I’m Still Standing’.
Now after having Kaftrio in September 2022 she and loyal partner Ciaran McVarnock welcomed their baby Colby Lee into the world.
On Tuesday Helen Knight, director of medicines evaluation at NICE, said: “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.
“Today’s decision follows NICE’s approval last year of Kaftrio and comes just weeks after Alyftrek was licensed for use in the UK.
“This is great news for people with cystic fibrosis and underlies our commitment to getting the best care to patients fast while ensuring the best value for the NHS drugs budget.”
The Daily Express has vowed to continue campaigning for UK patients who are, due to the rarity of their CF genes, as yet unable to take any of the current treatments.
Ludovic Fenaux, senior vice-president of Vertex International, said: “We’re pleased to have reached this agreement with NHS England that recognises the value that this new medicine brings to CF patients, their families and society.”
