Phenylketonuria results from the inability to metabolise phenylalanine. The treatment of phenylketonuria usually involves restricting dietary intake of phenylalanine to a small amount sufficient for tissue building and repair, in addition to phenylalanine-free L-amino acid supplementation.

Sapropterin dihydrochloride

• Soluble tablets 100mg

Notes

1. NICE TA729: Sapropterin (Kuvan) is recommended as an option for treating hyperphenylalaninaemia that responds to sapropterin (response as defined in the summary of product characteristics) in people with phenylketonuria (PKU), only if they are:
   1. under 18 and a dose of 10mg/kg is used, only using a higher dose if target blood phenylalanine levels cannot be achieved at 10mg/kg
   2. aged 18 to 21 inclusive, continuing the dose they were having before turning 18 or at a maximum dose of 10mg/kg
   3. pregnant (from a positive pregnancy test until birth)
2. Sapropterin is recommended only if the company provides it according to the commercial arrangement