Miracle drug that makes cystic fibrosis ‘vanish’ could soon be on NHS

Cystic fibrosis sufferers have revealed their thrill at a possible new NHS miracle pill – that makes most signs of the genetic disease simply ‘vanish’. 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.

Last summer – 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 now UK drugs regulator MHRA have approved the new Vertex pill Alyftrek and it’s so advanced, trials show patients no longer show up as having CF on vital ‘sweat tests’.

Now we can reveal this ‘next-gen’ drug could be on the NHS by late August if NICE (National Institute for Health and Care Excellence) – who are studying its cost-effectiveness – give medics the green-light to use it.

Excited CF patient and mother-of-one Carlie Pleasant, 35, told us: “This feels like one step closer to living lives without being reminded we even have CF – there’s a wonderful future on the horizon.

“When I first started campaigning with the Daily Express for access to Vertex’s CF drugs back in 2019, I was spending weeks away from my baby boy Jude in hospital with lung bugs.

“In 2020 after we secured the initial deals I went on Symkevi and then the first real wonder CF drug Kaftrio – which I’m on right now and have transformed my health and life.

“The last time I had a hospital admission was 2020. Now I am able to work, go on holiday, spend time with my husband and son and not have to worry about when the next chest infection is coming.”

Carlie was so instrumental in helping give CF campaigners a voice, the then Health Secretary Matt Hancock rang her to announce the first historic deal for Orkambi and Symkevi in 2019 – and again in summer 2020 for Kaftrio.

The Vertex drugs have rocketed the health of sufferers, with dying patients in intensive care that we urgently highlighted – now not only healthy but some 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 per cent 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 the Daily Express to say: “I’m so scared – I’m dying. What can I do?”​ – before weeping and trying to sing us ‘s hit ‘I’m Still Standing’.

Now after having Kaftrio she is not only engaged to loyal fiancé Ciaran McVarnock, in September 2022 the pair welcomed their baby Colby Lee into the world.

Carlie added: “It’s so exciting to think what other miracle health stories will be told if Alyftrek is approved by NICE this summer and starts to be rolled out.

“Our generation of sufferers are living experiments when it comes to CF treatments as in the UK the drugs have not been available for a relatively short time.

“Before I could never imagine a life without CF but Kaftrio changed all that. I feel so lucky – now my boy Jude is seven years-old and asks me ‘what is CF?’ as he is not used to seeing me poorly anymore.”

CF disrupts the transport of chloride across cell membranes so causes thicker, stickier mucus in the lungs and digestive system and higher levels of salty chloride – salt is sodium chloride – in sweat compared with those without CF.

A sweat chloride test, or sweat test, is the gold standard for diagnosing CF by measuring the amount of salt in sweat.

But phase 3 trials show people on Alyftrek have “carrier levels of sweat chloride”, meaning the pill has corrected the gene malfunction and it is acting like anyone without CF – so the key symptom has vanished.

Professor Alex Horsley, Professor of Respiratory Medicine at the University of Manchester, said that children and adults on Alyftrek was better in regard to “sweat chloride” compared to Kaftrio which could “translate to reduced risk of developing CF-related complications in the long term.”

Catherine Farrer, 41 – mum of CF sufferer Kate, 10 – told us she was thrilled when the Alyftrek MHRA news came through but is desperate for NICE to approve it and her daughter to have it.

Kate’s two CF genes do allow her to take Kaftrio but while one – the most common ‘508’ gene – makes her perfect for the drug her second gene is very rare and is only partially responding to the pill.

However trials of Alyftrek on patients also with that second rare CF gene have shown far better results.

The mum-of-two, of East Dulwich, South-East London, said: “Kaftrio completely changed the trajectory of Kate’s life, as she had had multiple admissions for intravenous antibiotics before then.

“She still suffers from chest infections, and unfortunately did have to have an admission for IVs last summer after multiple courses of oral antibiotics just didn’t clear up the unknown infection.

“Kaftrio has reduced her sweat test, however it falls within the ‘grey’ area now.

“Amazingly, her second CF gene mutation has been found to respond to Alyftrek, so you would expect her to get a bigger uplift in terms of the reduction in her sweat test.

“That would hopefully reduce the antibiotic burden and hopefully keep her out of hospital, therefore we are keen to try to switch her to Alyftrek when we can.

“Although, not a huge contribution to why we would switch her from Kaftrio, but the switch to a once-a-day dose, will also give her so much more freedom.”

Gymnast Kate – whose sister Orla, 7, does not have CF – still harbours dreams to reach the top in the sport and getting Alyftrek could boost those ambitions.

Catherine added: “She is competitive in artistic gymnastics, trampolining and tumbling – gymnastics is still her life and she does amazingly well for a kid with CF.

“She was the U11 Kent County level trampolining champion last year and was recently awarded a sports scholarship for entry into secondary school!”

The deal agreed last year between NHS England and Vertex only covered future licence extensions – when trials and tests show the drugs can be used for younger patients or those with rarer genes – for Orkambi, Symkevi and Kaftrio.

But although the deal did not include future drugs like Alyftrek, it did agree a pathway to easier negotiations between them for future innovative treatments.

One being looked at for future use is gene therapy to completely correct the faulty CF gene, which could finally help the around 10 per cent of global CF sufferers whose ultra-rare genes do not yet respond to Vertex’s drugs.

The Daily Express is committed to fighting until 100 per cent of sufferers have access, as around 1,000 British patients have those rare CF mutations.

A NICE spokesman told us: “NICE, NHS and Vertex are committed to work together on a path towards rapid access for all eligible patients for ALYFTREK and the treatment is in our work programme with an expected publication date for final guidance of 21 August 2025.”