Sapropterin for treating phenylketonuria: Appraisal consultation document

In DRAFT guidance, sapropterin is recommended as an option for treating hyperphenylalaninaemia in people with phenylketonuria (PKU), only if: they are under 18, a dose of up to 10 mg/kg is used, and the company provides it according to the commercial arrangement.

Source:

National Institute for Health and Care Excellence