sapropterin (Kuvan)

following a full submission assessed under the orphan medicine process:

sapropterin dihydrochloride (Kuvan®) is not recommended for use within NHSScotland.

Indication under review: the treatment of hyperphenylalaninaemia (HPA) in adults and paediatric patients of all ages with phenylketonuria (PKU) who have been shown to be responsive to such treatment.

In phase III studies in patients with a diagnosis of PKU, sapropterin was associated with a statistically significant reduction in blood phenylalanine concentration over placebo in one study, and statistically significant increases in phenylalanine tolerance when added to a phenylalanine-restricted diet compared with a phenylalanine-restricted diet alone in two other studies, one of which was placebo controlled.

The submitting company’s justification of the treatment’s cost in relation to its health benefits was not sufficient and in addition the company did not present a sufficiently robust clinical and economic analysis to gain acceptance by SMC.

This advice takes account of views from a Patient and Clinician Engagement (PACE) meeting.