Made by US firm BioMarin, the drug has been available for 12 years, but not in Britain. NICE, which approves drugs for the NHS, is proposing to let children have Kuvan but, to save cash, they must come off it at 18. Lily, 19, of West Drayton, Middlesex, said: “Limiting Kuvan just to kids is not right or ethical. It is vital Kuvan is accessible to all PKU sufferers – as the improvement to quality of life would be immense.”
A lifelong genetic condition, phenylketonuria leaves sufferers struggling to break down phenylalanine, an amino acid that is toxic to the brain. Kuvan helps PKU sufferers lead a normal life.
In her letter to the Health Secretary, Lily explained: “All of my life I have been on a highly restrictive low protein diet. I cannot eat meat, fish, eggs, dairy, pulses, and even some fruit and vegetables are restricted. If PKU sufferers don’t follow this diet the side effects are immense.”
Now health minister Lord Bethell has replied to Lily, stressing the NICE guidance may be “subject to change”.
He said: “Please be assured NICE only publishes final guidance after very careful consideration of the evidence.”
Over 240 sufferers wrote to NICE to give evidence of why PKU sufferers of all ages must be offered the drug.
Lily said: “Lord Bethell’s response gives me hope that NICE may change their decision.”
NICE expects to publish its final guidance in June.