How are you taking part in this consultation?

You will not be able to change how you comment later.

You must be signed in to answer questions

  • Question on Consultation

    Has all of the relevant evidence been taken into account?

  • Question on Consultation

    Are the summaries of clinical and cost effectiveness reasonable interpretations of the evidence?

  • Question on Consultation

    Are the recommendations sound and a suitable basis for guidance to the NHS?

  • Question on Consultation

    Are there any aspects of the recommendations that need particular consideration to ensure we avoid unlawful discrimination against any group of people on the grounds of race, gender, disability, religion or belief, sexual orientation, age, gender reassignment, pregnancy and maternity?
The content on this page is not current guidance and is only for the purposes of the consultation process.

Sapropterin for treating phenylketonuria

Closed for comments This consultation ended on at   Request commenting lead permission

2 Information about sapropterin

Marketing authorisation indication

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'.

Dosage in the marketing authorisation

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

Price

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).

The company has a commercial arrangement (simple discount patient access scheme). 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.