In June, the NHS agreed a deal with US firm Vertex for their pill dubbed “almost a cure” for the life-limiting genetic condition. But as thousands celebrated, some found to their horror their more rare CF gene mutation excluded them from taking it in Europe. The NHS can only prescribe drugs that are fully licensed and approved for use in Europe by the European Medicines Authority (EMA).
But now the Daily Express understands that around 700 more UK patients with those rarer mutations should now have Kaftrio in the New Year.
It comes after Vertex submitted fresh data to EU drugs appraiser, the EMA, on how those mutations respond to Kaftrio. Last night delighted father-of-two Stephen Walter, 44, said the news “means the world to him”.
The outdoor instructor, of Egremont, Cumbria, told us: “I have been poorly my whole life, it’s been a battle to stay healthy and strong.
“Kaftrio would give me an enormous boost and extra years of my life that I could spend with my two children. When the June NHS deal was done, I typed my rare mutation into Vertex’s website in the US and it said I could take Kaftrio.
“But when the list of EU-approved CF mutations was published last month mine was missing, so legally the NHS can’t let me have it. I don’t want to tempt fate but if this is sorted in the New Year it would be incredible. It will change my life and give me new life.”
700 more UK patients with those rarer mutations should now have Kaftrio in the New Year
There are 10,509 people in the UK with CF, which slowly destroys the lungs, with sticky mucus, and the digestive system.
Every human has two copies of the CF transmembrane conductance regulator (CFTR) gene but you must inherit two mutated CFTRs to be born with CF.
The most common mutation is called F508 and if you have two, you can take Vertex’s Orkambi, Symkevi and Kaftrio drugs.
Having two F508 mutations accounts for approximately half of the UK’s CF population.
But super advanced Kaftrio is also designed to work for anyone with just one 508 gene and any other second rarer mutation – accounting for 90 percent of all CFers.
The day I have Kaftrio will be one of the best in my life
A mother diagnosed with CF aged 36 is desperate to take wonder drug Kaftrio as soon as she can.
Sarah Wooley, 40, was told by doctors growing up she had severe asthma and was just very prone to sinus and chest infections.
In fact, the mum-of-two was born with cruel genetic condition CF that was only spotted four years ago by an eagle-eyed doctor.
But then Sarah, of Stoke, was told there was no drug – not even Kaftrio – that the NHS was legally allowed to give her.
Now the fashion worker has told of her excitement that possibly early next year she could be on the wonder pill. She added: “I’ve always known I was poorly but not that it was actually cystic fibrosis until four years ago.
“Looking back it is hard to believe for all those years no doctor realised I had CF. I was always on and off antibiotics.
Sarah Wooley and family can look ahead more positively
“I was always having sinus problems and had a terrible smoker’s cough. A real hacking cough.”
When Sarah – mum to Charlotte, 13, and George, nine, and married to Grant, 41 – was finally told she had CF she was shattered.
She burst into tears and admitted: “I was not in a good place. I was thinking: ‘Am I going to see my kids grow up?'” When an NHS deal for Kaftrio was agreed last June she assumed she would be able to take it, like those with her mutations in the US.
But she was devastated when told the restrictions of the EU licence meant she could not yet have it. Now she hopes that by early 2021 she will be able to access it on the NHS, as her mutation should by then be covered.
She said: “When I saw others start to get it I was delighted for them but sad. I’m trying not to let myself dream but the day I have Kaftrio will be one of the best in my life.”
We just want what everyone else has… the chance to live a full and happy life
Thrilled Carl Butler dreams that CF will soon stop dominating his life after once coughing so hard as a child, he suffered a hernia.
Carl, 34, loves spending time with partner Jennifer, 35, and their 21-month-old son Nicholas at home in Greenwich, south-east London.
The accountant keeps as healthy as he can but having nasty lung bacteria mycobacterium abscessus often leaves him exhausted. And then he has no energy to play with his little boy they had through IVF, as most men with CF are unable to have children naturally.
Carl Butler with partner Jennifer and son Nicholas
Since 2012 he’s been lucky enough to take US firm Vertex’s first CF drug Kalydeco, that has kept him stable.
But next year he hopes to get the incredible “triple” pill Kaftrio – which could transform his life forever.
He said: “I’m grateful to have had Kalydeco all these years and felt guilt at first as only a fraction of CFers were able to take it. When I realised I would not be able to yet take Kaftrio I was so happy for everyone else but gutted. It was hard knowing people right now in the US who have my mutations can take Kaftrio but here we can’t.
“Now we just want what everyone else has – the chance to live a full and happy life and Kaftrio, I hope, can do that for me. I want to not only see my boy grow up but maybe my grandchildren, too. It’s something most people take for granted.”