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Judge forces NHS England rethink on Kuvan

The health service claims the drug is not clinically effective

NHSA judge has ordered NHS England to reconsider after refusing to fund treatment with Biomarin's Kuvan for a boy with the rare genetic disorder phenylketonuria (PKU).

The unnamed seven-year-old boy also has severe autism, which makes adhering to the dietary restrictions usually deployed to manage PKU - and prevent the serious brain damage that can be caused by the condition - difficult to achieve.

PKU stems from a deficiency of the enzyme phenylalanine hydroxylase (PAH), which causes levels of the amino acid phenylalanine to accumulate in the body, in turn causing neurological damage. Keeping phenylalanine out of the diet can prevent that happening as the body doesn't produce the amino acid itself.

BioMarin won EU approval in 2008 for Kuvan (sapropterin dihydrochloride), a drug that is used alongside a phenylalanine-restricted diet to reduce levels of amino acid even further, but so far the NHS has refused to fund the treatment, which costs around £100 per day.

The lawsuit said that because his autism made it impossible to maintain a diet low in phenylalanine, for example by avoiding eggs, meat, fish and cheese, the boy should be considered a special case and be granted access to the drug.

After two years of deliberations the judge in the case has sided with the family and ordered the NHS to reconsider use of the drug under the individual funding request (IFR) process. The boy's counsel, Ian Wise QC, said that funding had been repeatedly refused by NHS England with differing reasons given.

Mrs Justice Andrews ordered the rethink because one of the reasons for denying the drug given by the NHS - that Kuvan was not clinically effective - was not supported by the evidence, but she stressed the outcome of the matter was far from certain.

"Whilst this judgment is bound to give rise to a degree of optimism, I must caution against raising hopes too high," she said. "The fact that this claim for judicial review has succeeded does not mean that there will necessarily be a favourable outcome to this IFR application."

"We note that the judge accepted that NHS England had met its statutory duties under the Children Act and rejected the assertion that NHS England was acting in breach of the Human Rights Act," said NHS England in a statement.

"While the outcome of this case is limited to the particular circumstances of this funding request and does not have any wider implications for how NHS England makes decisions regarding the funding of treatments, we will of course take a further look at the IFR in question in the light of any further information provided to us."

Kuvan was once tested as a treatment for autism itself, but failed to have an impact on the symptoms of the condition.

Article by
Phil Taylor

9th August 2017

From: Healthcare

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