Sapropterin for treating hyperphenylalaninaemia in phenylketonuria

2.1 Sapropterin (Kuvan, BioMarin) is indicated 'for 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'.

2.2 The dosage schedule is available in the summary of product characteristics.

2.3 The list price of sapropterin is £597.22 per 30‑tablet pack. Each tablet contains 100 mg sapropterin dihydrochloride (excluding VAT; BNF online, accessed January 2021).

2.4 The company has a commercial arrangement. This makes sapropterin available to the NHS with a discount. The size of the discount is commercial in confidence. It is the company's responsibility to let relevant NHS organisations know details of the discount.