Since 2019 the Daily Express has fought for access to US firm Vertex’s incredible drug Kaftrio, hailed as “almost a cure for CF”. Around 10,500 people in the UK have CF, a genetic illness that clogs the lungs with mucus and affects the pancreas, liver, kidneys and bowel. Only half of the sufferers live to 32. Last summer the NHS struck a deal for the wonder pill, meaning over 90 percent of those aged 12 and over have access. And now Vertex has finally applied to the UK drug regulator, the Medicines and Healthcare products Regulatory Agency, to give Kaftrio to the UK’s 1,300 suitable youngsters aged six to 11.

Vertex’s executive vice-president Nia Tatsis said: “Today’s news is an important milestone in broadening our access worldwide.”

It’s hoped MHRA will rubber-stamp approval by the autumn, which could dramatically change the lives of young CF sufferers.

Dozens of those excited youngsters are brave, crusading sufferers who joined the Daily Express in our CF fight.

In 2019, when Tristan Scott-Binns was nine, he wrote to Dr. Jeff Leiden, the then-CEO of Vertex, begging for the life-saving treatment.

He revealed he lost his uncle Adam, 25, to CF and his dad Andrew from depression, leaving Tristan to be raised by his grandparents. Dr Leiden was so moved by his note he vowed he “wouldn’t rest” until the UK has access to the medications.

Now 11, Tristan is on Vertex’s older drug, Orkambi – but hopefully just months away from starting Kaftrio.

Grandad John, 72, of Holmfirth, West Yorks, said: “This is fantastic news. CF has had a devastating effect on our family and so many others. Thank you Daily Express and everyone who’s made this possible.”

Five-year-old Darcie Kiszka often asks her mum, Marta: “Who gave me CF? I don’t want to be special.”

Marta, 35, of Canterbury, Kent, said: “Now we know our daughter will get that chance to live longer, we are crying with happiness.” The rare CF genes of five-year-old Theo Powell, of Tunbridge Wells, Kent, mean he is unable to take Orkambi.

His parents, the Rev Laurence Powell and Caroline, still campaigned with the Daily Express for the drugs.

Now Mr Powell, 34, has said: “We have been so eager for Theo to get on Kaftrio. It has been out there for over a year and we have patiently waited his turn.”

Post source Daily Express

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