Remit: To appraise the clinical and cost effectiveness of sapropterin within its marketing authorisation for treating phenylketonuria.
Status In progress
Process STA 2018
ID number 1475

Provisional Schedule

Expected publication 18 August 2021

Project Team

Project lead Thomas Feist

Email enquiries

Evidence Review Group / Assessment Group Liverpool Reviews and Implementation Group, University of Liverpool


Companies sponsors BioMarin
Patient carer groups Metabolic Support UK
  The National Society for Phenylketonuria
Professional groups British Inherited Metabolic Diseases Group
  Royal College of Pathologists
  Royal College of Physicians


General commentators All Wales Therapeutics and Toxicology Centre
  British National Formulary
  Department of Health, Social Services and Public Safety for Northern Ireland
  Healthcare Improvement Scotland
  Medicines and Healthcare Products Regulatory Agency
  Scottish Medicines Consortium
  Welsh Health Specialised Services Committee


Key events during the development of the guidance:

Date Update
08 June 2021 Committee meeting: 3
20 April 2021 The appraisal was discussed by the technology appraisal committee on 7 April 2021. The discussion was adjourned, to be resumed at a further meeting. The date for next discussion is Tuesday 8 June 2021.
07 April 2021 Committee meeting: 2
25 February 2021 - 18 March 2021 Appraisal consultation
02 February 2021 Committee meeting: 1
29 May 2020 Invitation to participate
07 April 2020 In progress. Topic is in progress
07 April 2020 This appraisal topic was suspended during 2019 as the company BioMarin withdrew from the technology appraisal process. NICE have continued liaison with the company and NICE are now able to inform you that BioMarin have now re-engaged in the appraisal process. Therefore, this appraisal has now been rescheduled back into the work programme and is anticipated to begin during late May 2020.
31 October 2019 Suspended. Following recommencement of the appraisal and a company update, this appraisal topic has been suspended because BioMarin has withdrawn from the technology appraisal process. NICE will continue to monitor any development and provide further information, as available.
19 August 2019 In development. This appraisal topic recommenced at ‘clarification stage’.
30 April 2019 In development. This topic will be rescheduled into the technology appraisal STA programme and will recommence at the ‘clarification stage'.
31 January 2019 Suspended. Following a challenge to the decision to review sapropterin for treating phenylketonuria through the technology appraisal programme, NICE has made the exceptional decision to pause the appraisal at this point and ask the topic selection decision making panel to reconsider whether this topic is suitable for the NICE highly specialised technologies or technology appraisal programme. After this a formal referral will be required from the Minister to determine the process under which sapropterin will be assessed. An update will be provided once a formal referral is received.
16 October 2018 Invitation to participate
09 October 2018 In progress. Topic referred
13 August 2018 - 10 September 2018 Consultation on suggested remit, draft scope and provisional matrix of consultees and commentators

For further information on how we develop guidance, please see our page about NICE technology appraisal guidance