Pill helps 90 per cent of patients
CYSTIC fibrosis is a genetic condition that clogs the lungs with sticky mucus, and also affects the pancreas, liver, kidneys and bowel.
There are around 70,000 sufferers in the world – 10,500 in the UK – but only about half live to the age of 32.
Sufferers spend hours a day doing physio to clear their lungs, and must take digestive enzymes with every morsel of food to absorb any calories.
Since our first CF crusade, all UK sufferers have access to Vertex’s Kalydeco, Orkambi and Symkevi.
The three medicines help reverse CF’s deadly effects but only help around 50 per cent of sufferers, as the rest have rarer CF gene mutations.
But the upcoming Trikafta – or Triple Therapy – pill helps 90 per cent of sufferers.
It is no cure for CF but this drug is the closest to being one.
There is currently no available treatment for the 10 per cent of sufferers Trikafta does not cover.
Hailing the new Triple Therapy, Vertex’s outgoing CEO Dr Jeff Leiden said: “When I started at Vertex in 2011, I promised we would not stop until we had a treatment for nearly everyone with CF.
“This is a great way to end a career. We have conquered a disease. You don’t get to do that very often in this business.”
THE DAILY Express today launches a new cystic fibrosis campaign for an upcoming drug branded “almost a cure” for the cruel condition.
Since February your crusading newspaper has fought for CF sufferers to get access to life-changing drugs Symkevi and Orkambi.
We shattered a stalemate between the NHS and US pharmaceuticals firm Vertex and helped them find a historic compromise deal.
But current drugs help just 50 per cent of sufferers – and can only “stop the clock” on CF and prevent sufferers from getting worse.
Vertex’s latest CF medicine, Trikafta, is currently being tested for an EU licence.
Trikafta, also known as Triple Therapy, is able to help 90 per cent of patients and could be available as early as June.
One UK patient trialling Trikafta stunned the world by revealing how it had boosted his lung function from
BATTLE FOR NEW WONDER DRUG
30 per cent to 90 per cent.
When Orkambi was licensed it took four years for Vertex and the NHS to agree a deal, which they did last month.
Today we are urging NHS, Vertex and UK drugs appraiser the National Institute of Health and Care Excellence (NICE) to begin a deal now – so Trikafta can be rolled out as soon as possible.
CF sufferer and Daily Express writer Carlie Pleasant, 29, said it could give her decades longer with her two year-old son Jude and husband Chris.
Carlie, of Sittingbourne, Kent, said: “I have access to Orkambi and Symkevi but Trikafta is the cutting edge of CF treatment.
“I’ve been following the results of CF patients in America already on it and the results are exciting.
“It will improve my quality of life even more. And for those who cannot take the current drugs available it is essential that they have access as soon as possible.”
Alison Dillon, 53 – who helped organise the LayDown4CF protest outsideWestminster in July – is mum to CF-suffering TV-actor son Oli, 21.
Cough
She told us: “I am so pleased that the Daily Express is sticking with us for the next phase of our campaign.
“Orkambi and Symkevi are lifesaving medicines but Trikafta is truly life-changing.
“Oli is 21 and has had a number of infections this year. I have seen stories of those taking Trikafta who have turned their health around who
now barely cough. I want that for Oli and believe with Trikafta he will have the closest thing there is to a cure and a normal life.”
Around June next year Trikafta is expected to start an 18-month data gathering process in the UK.
But Vertex, unhappy with NICE’s way of evaluating its precision drugs, has vowed not to submit Trikafta for NHS appraisal until January 2021.
Unless the situation changes, the drug would not be available on the NHS until around September 2021 at the earliest.
Our Cystic Fibrosis Wonder Drug Now campaign is calling on all sides to speed up this process.
Campaigners are also pleading
for an interim deal so patients can immediately start to take Trikafta the moment it is licensed next summer.
Sharon Cranfield, 54, is one of the UK’s leading CF campaigners and desperately wants Trikafta for her poorly daughter Jessica, 18.
The mum, of Tadworth, Surrey, told us: “It’s essential Trikafta is available immediately EU approval is received.
“Every day counts and any delay will deny my daughter the chance to have a drug that will transform her life. She has so much lung damage already and is so vulnerable.”