Thank you! Joy of PKU sufferers as we lead drug fight
SUPPLY KUVAN ON THE NHS
DELIGHTED PKU sufferers have hailed the Daily Express after we launched a crusade to secure them a life-changing drug.
Genetic condition phenylketonuria leaves sufferers struggling to break down phenylalanine (Phe) – an amino acid that comes from protein.
Phe is toxic to the brain at high levels. For this reason they must live on a severely restricted diet.
US firm BioMarin’s drug Kuvan, which could help 600 UK sufferers lead a normal life, has been available in the UK for 11 years – but not on the NHS.
On Saturday we launched a new campaign to demand BioMarin and the NHS thrash out an immediate deal for the drug after talks failed over pricing.
And families at the annual National Society for Phenylketonuria conference in Ashford, Kent, rushed to snap up copies of the paper – and posted a video shouting, “Thank you Daily Express”.
Siblings Holly and Callum Graham, of Godalming, Surrey, both have PKU and vowed to support our health justice crusade.
Callum, 26, said: “I’m so grateful you are fighting with us. I hope this will help us get a solution at last.”
PKU sufferer Heidi Sudworth, 38, came to the conference from Swindon with PKU-free five-year-old daughter Anya Jayne.
Heidi told us: “I’ve struggled with my PKU for my whole adult life. It’s so hard to cope every day.”
Kuvan, available here since 2008, reduces levels of Phe but has yet to be approved by NHS drugs appraiser, the National Institute for Health and Care Excellence (Nice).
It costs around £45,000 a year per adult and around £4,000 for a child.
NHS England wants BioMarin to re-engage with the Nice process after the firm withdrew last October.
But BioMarin says the NHS wants an unrealistic 80 per cent discount.
BioMarin told us there is “no realistic probability” of getting a deal done while the NHS insists on the massive markdown.
The firm told the Daily Express: “BioMarin has offered the lowest tier of pricing in the world to NHS England – but the NHS does not recognise the burden and severity of phenylketonuria.”
Fighting… Evelina with son Derren, who struggles with the condition. Right, families hold aloft copies of the Daily Express at the PKU conference
EVELINA Hudson used to work as a translator but had to give it up to care for seven-year-old Derren.
Derren was born with PKU but his non-identical twin brother George is PKU-free. The pair have a 10-year-old brother and a sister aged nine.
Frustrated Evelina, 43, said Kuvan would make a huge difference to the diet and mental health of Derren, who has to take foul-tasting synthetic protein supplements.
She said: “I cannot work because of the emotional needs and physical food care required by Derren.
“If he continues without Kuvan, I fear he will suffer mental health issues.”
The single mum, from Sutton, south London, spends much of her day cooking separate meals for her family.
Evelina said: “His most common emotional state about food is hate and depression – already, at this young age.
“Kuvan would help Derren to finally enjoy food and feel good. It would bring some normality and I could feel I can return to work.”