Too much protein causes brain damage so they must avoid meat, fish, eggs, dairy and instead endure foul-tasting synthetic food, fruit and some vegetables. For a year the Daily Express have urged drugs approver NICE (National Institute for Health and Care Excellence) to recommend Kuvan on the NHS to ease sufferers’ agony. But in a cruel victory NICE has finally approved the drug but – for “cost effective” reasons – only for children. It means all adults must continue to suffer and even those children to get Kuvan will be stripped of it on their 18th birthday.
Furious campaigner Kate Learoyd told us: “NICE’s decision is unrealistic and unfair and we will be challenging it.
“People with PKU are not cured of their condition on their 18th birthday – this condition stays with you for life.
“The idea of the NHS taking a teenager’s medicine away from them on their 18th birthday is simply cruel.”
Caroline Graham, 55, has two grown-ups with PKU desperate for Kuvan – architectural assistant Callum and music student Holly.
She raged: “NICE are setting children up to fail by giving them Kuvan now, then taking it off them at 18.
“Little children with PKU grow into adults with PKU. A lifelong diagnosis should have a lifelong treatment.
“One of the most difficult times to manage PKU is when you turn 18, often leave home and have to survive without close family support.
“Taking away their medication then makes no sense at all.”
Devastated Callum, 26, said: “I can’t believe anyone thinks giving a child a drug and then removing it on their 18th birthday is moral.
“This disorder does not diminish over time, it stays the same. PKU for me has been a life sentence, not just a childhood problem. This is immoral and wrong.”
While tearful Holly, 24, told us: “I work extremely hard to keep on top of my PKU but fail often.
“It’s not just my present affected by this decision but my whole future. I have always wanted children since I was little.
“Now more than ever I am highly doubtful and that scares me.”
There are around 2,000 people with PKU in NHS care in England alone but Kuvan is estimated to only be suitable for around 600 of them.
Since 2008 Kuvan, US firm BioMarin’s brand name for the drug sapropterin, has been available in Europe and in use in over 50 poorer countries including Croatia.
It costs around £45,000 per adult-per-year but as low as £4,000 for a toddler yet for over a decade the NHS have failed to strike a deal.
In 2021 BioMarin’s patents expired, meaning now rival firms can offer the UK their own, cheaper version – but these have yet to be approved here.
Yesterday NICE told the Daily Express they acknowledged that sapropterin (Kuvan) “could prevent long-term irreversible brain damage in children”.
But they claimed it was not “an effective use of NHS resources” for adults as dosage is based on weight, so they need more.
NICE also claimed there was an “absence of data” on whether sapropterin might “improve quality-of-life” for adults and even boldly claimed: “There is also no risk of irreversible brain damage in adults with PKU.”
Yet their very own consultation document contradicts that, conceding “brain development does not stop until around age 25.”
Their decision is open for public consultation for a month before it is rubber-stamped in April. Children could start to access Kuvan after June.
NICE’s deputy chief executive Meindert Boysen told us they are recommending Kuvan for children but the NHS should stop funding it when they turn 18.
He told us: “The benefit for adults is not one that we think worth paying for. It would mean (children turning 18) would have to stop.
“There is a discussion to be had with the clinician and the patient about what the best timing is, but that’s our guidance.
“There will be individual circumstances to continue for a bit longer but we would not be able to make NHSE pay for it after that time.”
In a brief statement, NHS England claimed they were powerless to act over Kuvan as they are beholden to the judgements of NICE.
An NHS spokesperson said: “NICE recommendations are legally binding and the NHS will implement their final decision”.
However NHSE have held four reviews into making Kuvan available – in 2015, 2018, 2019 and 2020 – and each time chosen themselves not to make the drug available.