LIFE-CHANGER!
Victory in wonder drug battle
YOUNGSTERS with hellish genetic condition PKU claimed victory yesterday, following the Daily Express campaign for a life-changing drug.
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 per cent.
Unfair
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.”