Alyftrek is so advanced that trials show patients no longer exhibit CF symptoms in vital tests.
The “next generation” drug has been backed by regulators and could be available on the NHS by late August if a medicines watchdog gives doctors the green light to use it. Catherine Farrer, 41, mother of CF sufferer Kate, 10, said she was thrilled by the Alyftrek approval and is desperate for her daughter to have it. She added: "We are keen to try to switch her to Alyftrek when we can."
CF is a life-limiting genetic condition that affects 11,000 people in the UK, but before 2019 the NHS had no effective treatment.
However, last summer - after a five-year Daily Express campaign - US drug giant Vertex agreed a health service deal so that 90% of CF patients suitable for its treatments - Orkambi, Symkevi and Kaftrio - could access them.
Bosses at watchdog the National Institute for Health and Care Excellence are now studying the cost-effectiveness of Vertex pill Alyftrek. CF patient Carlie Pleasant was also delighted that the Medicines and Healthcare products Regulatory Agency had backed the new drug.
She said: "This feels like one step closer to living lives without being reminded we even have CF - there's a wonderful future on the horizon."
The mum of one, 35, added: "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. They have transformed my health and life.
"The last time I had a hospital admission was in 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 for Kaftrio in 2020.
She 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 because in the UK the drugs have 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 any more."
The Vertex drugs have transformed the health of sufferers.
Patients are no longer struggling in intensive care but are healthy and some have started families.
Nicole Adams, 28, was fighting for every breath in Belfast City Hospital's intensive care unit in 2019.
Just 10% of her lungs were working and her tearful loved ones prepared to say their 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?" She then wept and tried to sing Elton John's hit I'm Still Standing.
Now, after having Kaftrio, she and fiance Ciaran McVarnock, 27, welcomed their baby Colby Lee into the world in September 2022.
CF disrupts the transport of chloride across cell membranes which causes thicker, stickier mucus in the lungs and digestive system and higher levels of salty chloride in sweat compared with those without CF. A sweat chloride test is the gold standard for diagnosing CF by measuring the amount of salt in the moisture. But phase three trials show people on Alyftrek have "carrier levels of sweat chloride".
image [https://cdn.magzter.com/1583504377/1742173784/articles/xtcl3ERrFKBNlYt4tesys/hfAEs8ktW1742185566498.jpg]
This means that the pill has corrected the gene malfunction and it is acting like anyone without CF - so the key symptom has vanished.
Alex Horsley, professor of respiratory medicine at Manchester University, said the results for children and adults on Alyftrek were better in regard to "sweat chloride" compared with Kaftrio.
He explained it could "translate to reduced risk of developing CF-related complications in the long term". Mrs Farrer explained that daughte...
The “next generation” drug has been backed by regulators and could be available on the NHS by late August if a medicines watchdog gives doctors the green light to use it. Catherine Farrer, 41, mother of CF sufferer Kate, 10, said she was thrilled by the Alyftrek approval and is desperate for her daughter to have it. She added: "We are keen to try to switch her to Alyftrek when we can."
CF is a life-limiting genetic condition that affects 11,000 people in the UK, but before 2019 the NHS had no effective treatment.
However, last summer - after a five-year Daily Express campaign - US drug giant Vertex agreed a health service deal so that 90% of CF patients suitable for its treatments - Orkambi, Symkevi and Kaftrio - could access them.
Bosses at watchdog the National Institute for Health and Care Excellence are now studying the cost-effectiveness of Vertex pill Alyftrek. CF patient Carlie Pleasant was also delighted that the Medicines and Healthcare products Regulatory Agency had backed the new drug.
She said: "This feels like one step closer to living lives without being reminded we even have CF - there's a wonderful future on the horizon."
The mum of one, 35, added: "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. They have transformed my health and life.
"The last time I had a hospital admission was in 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 for Kaftrio in 2020.
She 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 because in the UK the drugs have 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 any more."
The Vertex drugs have transformed the health of sufferers.
Patients are no longer struggling in intensive care but are healthy and some have started families.
Nicole Adams, 28, was fighting for every breath in Belfast City Hospital's intensive care unit in 2019.
Just 10% of her lungs were working and her tearful loved ones prepared to say their 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?" She then wept and tried to sing Elton John's hit I'm Still Standing.
Now, after having Kaftrio, she and fiance Ciaran McVarnock, 27, welcomed their baby Colby Lee into the world in September 2022.
CF disrupts the transport of chloride across cell membranes which causes thicker, stickier mucus in the lungs and digestive system and higher levels of salty chloride in sweat compared with those without CF. A sweat chloride test is the gold standard for diagnosing CF by measuring the amount of salt in the moisture. But phase three trials show people on Alyftrek have "carrier levels of sweat chloride".
image [https://cdn.magzter.com/1583504377/1742173784/articles/xtcl3ERrFKBNlYt4tesys/hfAEs8ktW1742185566498.jpg]
This means that the pill has corrected the gene malfunction and it is acting like anyone without CF - so the key symptom has vanished.
Alex Horsley, professor of respiratory medicine at Manchester University, said the results for children and adults on Alyftrek were better in regard to "sweat chloride" compared with Kaftrio.
He explained it could "translate to reduced risk of developing CF-related complications in the long term". Mrs Farrer explained that daughte...
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Daily Express (Digital) - 1 Issue, March 17, 2025