A drug for phenylketonuria is to be made available to pregnant women and young adults, it has been announced.
The drug sapropterin helps patients control the condition – normally managed through a protein-restricted diet – and was to have been offered to children.
In final guidance, the drug will be offered to pregnant women to help prevent neurological damage to their unborn children, the National Institute for Health and Care Excellence said.
It will also be offered to patients up to the age of 22 to help protect the brain in the final stages of development, according to the new guidance.
NICE said the drug remained too expensive to justify its use on adult patients.
The NHS currently treats 2,000 patients with PKU. About 75 children under the age of 18 would receive the drug.
Deputy chief executive Meindert Boysen said: “Although we’re pleased to now be able to recommend sapropterin for people up to 22 years of age and pregnant women, it’s disappointing not to have been able to extend the recommendation to all adults.
“Even when considering some additional potential benefits of sapropterin in this group, the price of the drug was too high to allow it to be considered an acceptable use of NHS resources. The committee were aware that 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.”
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