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For 13 years, US firm BioMarin’s drug Kuvan has eased the agony of people across Europe who must avoid protein for fear of suffering brain damage. Despite England and Wales having just 2,000 PKU patients, it has not been accessible here.
Sufferers endure a brutal regime of prescription, non-protein synthetic food, avoiding meat, fish, eggs, dairy and many fruits and vegetables.
For 18 months this newspaper has urged the NHS and drugs adviser the National Institute for Health and Care Excellence (Nice) to ease patients’ pain.
Yesterday, Kuvan ‑ BioMarin’s brand name for the drug sapropterin ‑ was approved for England and Wales…but, in a bittersweet decision, only for those under 22 and mums-to-be.
However, there is hope for the future. Many of BioMarin’s patents expired in January, which means rivals can license generic drug copies.
NHS England (NHSE) now hopes talks with pharmaceutical firms will lead to all sufferers being able to access the drug.
Campaigner Kate Learoyd, whose son Alex has been on Kuvan for seven years through a clinical trial, said yesterday: “I am so happy and relieved that children and young people will start taking Kuvan soon and I would like to thank the Daily Express for campaigning alongside us.”
But she warned: “PKU does not disappear on your 22nd birthday.
“Cheaper generic versions offer the solution. It’s essential that the NHS gets a deal for all.”
NHSE and the NHS in Wales have 90 days to make Kuvan available. Northern Ireland and Scotland have their own processes.
People with PKU ‑ the abbreviation for phenylketonuria ‑ cannot properly digest the amino acid phenylalanine. Amino acids, the building blocks of protein, are broken down by the body, but in people with PKU the levels build up and can cause brain damage.
Clinical trials prove Kuvan reduces phenylalanine levels for many and Nice evidence shows it lowers the need for low-protein foods and supplements by 71.2 percent.
That means sufferers can eat out, cook with more ingredients and have fish, meat and dairy in their diet.
NHSE has held four reviews into Kuvan since 2015. Despite the drug being licensed for the UK since 2008, Nice waited 10 years before starting to investigate.
A preliminary decision was reached this year, to offer it to under-18s. After heavy criticism, it included pregnant sufferers and those aged 21 and under.
Meindert Boysen, of Nice’s Centre for Health Technology Assessment, said: “The price of the drug was too high to allow it to be considered an acceptable use of NHS resources.
“The committee was aware generic products could be available in the near future and hoped these would be priced to allow access to this drug for all adults with PKU.”
Former health secretary Jeremy Hunt told the Daily Express: “It is not fair to exclude individuals from this treatment. They have already waited too long.”
Cheaper versions offer optimism
Rival firms like Swiss-based Dipharma can now make and offer a cheaper version of Kuvan.
The Daily Express understands Dipharma’s sapropterin is being reviewed by the Medicines and Healthcare products Regulatory Agency. In a possible future deal, it could be licensed by around October.
Campaigner Caroline Graham, 55, of Godalming, Surrey, whose children Holly, 24, and Callum, 27, have missed out on getting Kuvan due to their age, said: “My children have their whole lives ahead of them. “They are too young to be written off.”
Joy for campaigner who pleased with PM
A PKU sufferer can finally access Kuvan after her pleas in the Daily Express to the Prime Minister helped secure the landmark deal.
Lily Wood, 20, wrote and spoke to Boris Johnson and health ministers many times, asking them to prioritise the drug.
In June, after hearing Nice planned to only let under-18s use it, she wrote again to the PM in our paper and weeks later the maximum age was lifted to 21.
Lily, of West Drayton in Middlesex, told us: “I can’t believe it ‑ it has been such a long fight for me but far longer for others. I cried when we heard the news, tears of happiness but it’s bittersweet as so many sufferers are missing out.
“Living with PKU is so much harder than anyone can realise ‑ it’s not just a case of enduring a tough diet. It dominates your life.”
Parents Chris, 57, and Hayley Wood, 45, were shocked when Lily was born with PKU as they had never heard of the disorder.
Forced to stick to prescribed bread with fruit or vegetables, Lily can only eat the protein equivalent of half a chicken nugget a day and must carefully weigh every snack.
Stanley gives thumbs-up to breakthrough
Stanley Brown gives the news that he might soon be on wonder drug Kuvan a big thumbs-up.
The six-year-old struggles to find any non-protein food he likes and, heartbreakingly, has told his parents Jessica and Matt: “I wish I didn’t have PKU!”
But delighted Jessica, 39, said yesterday: “We’re thrilled this drug will be made available to Stanley after such a long, long wait. We are so eager to get started with it and hope after this announcement things can move quickly.
“But we’re apprehensive that at some point in his life the drug will be withdrawn and feel terrible for those people that won’t be able to access it.”
Last year Jessica, of Halesowen, West Midlands, spoke of the stress of PKU dominating family life.
“We cannot eat out often and if we do we need to do extensive research. We also have to make sure he doesn’t eat his friends’ food or treats ‑ which makes him feel especially different.”
Stanley has to avoid meats, nuts, fish, dairy products ‑ and even some vegetables. Jessica, a teacher, added: “Kuvan would give him the freedom to enjoy food and take away a huge level of stress.”
Hope for Willow
Willow Hodder is jumping for joy because soon she may be able to eat the same food as her big sister.
Three-year-old Willow cannot enjoy what Grace, eight, has as PKU limits her to 3g of protein a day.
Meat, eggs, wheat and dairy products are all banned.
Mum Chelsea Glover, 28, said it was “gruelling” measuring every meal and morsel.
But if blood tests show Willow will respond to Kuvan, she hopes her daughter can enjoy tasty food.
Chelsea, of Bromborough, on the Wirral, said:”Kuvan will give sufferers the freedom to live like you and I. Willow could be able to enjoy the same lunch from the canteen as her peers.
“Willow starts nursery at a primary school in September and the preparation has been lengthy.”
Chelsea’s partner Stewart, 32, also has PKU, although he is not on a special diet.
Chelsea said Willow wants to eat like Grace.
Willow’s mum added: “She doesn’t understand why she can’t and the guilt is setting in for me.”
Comment by Anita MacDonald
I have been looking after children with PKU for more than 30 years. The clinic at Birmingham Children’s Hospital has more than 100 with the condition in our care.
The standard treatment for PKU is an extremely restrictive diet and this needs to be adhered to in order to prevent damage to the brain.
It is very hard for the children and their families to live with the constant strain and worry about their diet.
I am, therefore, absolutely delighted that we can offer Kuvan to our patients.
I have been involved in several clinical trials for the drug and I have seen that Kuvan treatment can make a life-changing difference for the children and their families.
The youngsters take the pills dissolved in water and drink with their breakfast. Those who respond to treatment can then keep their levels of the essential building-block protein phenylalanine within safe limits while having a more liberalised diet containing more protein.
This means the children can enjoy many normal foods that everyone else would take for granted.
Anita MacDonald is a consultant dietitian
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