Sapropterin (Kuvan®) is a drug manufactured by the pharmaceutical company Merck Serono, which is useful in some variants of PKU, usually in addition to dietary treatment. In fact a small number of Irish patients were involved in the original clinical trial for this medication here at NCIMD.

This medication is currently not funded by the HSE, as an assessment by the National Centre for Pharmacoeconomics (www.ncpe.ie) in 2009, concluded that Kuvan was not a cost effective treatment.

We, at NCIMD, support a re-assessment by Merck Serono and the Department of Health/HSE for its use in the Irish setting. Realistically, it will not be suitable for most Irish PKU patients, but there is a small minority of patients where a treatment response may be expected.

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