Suggested remit: To appraise the clinical and cost effectiveness of pegzilarginase within its marketing authorisation as an add-on treatment for arginase-1 deficiency.
Status In progress
Technology type Medicine
Decision Selected
Reason for decision Anticipate the topic will be of importance to patients, carers, professionals, commissioners and the health of the public to ensure clinical benefit is realised, inequalities in use addressed, and help them make the best use of NHS resources
Further information The topic routing was discussed at the Topic Selection Oversight Panel (TSOP) in April 2023. The panel concluded that the topic was suitable for Highly Specialised Technologies. Please see project documents for further details. The evaluation is expected to begin with an invitation to participate in September 2023, with a first committee meeting in May 2024.
Process HST Standard
ID number 4029

Provisional Schedule

Committee meeting: 1 15 August 2024
Expected publication 30 October 2024

Project Team

Project lead Vonda Murray

Email enquiries


Companies sponsors • Immedica
Others Department of Health and Social Care
  NHS England
Patient carer groups Gene People
  Metabolic Support UK
Professional groups Royal College of Physicians
Associated guideline groups NICE - National Guideline Alliance
  NICE - National Guideline Centre
Associated public health groups National Institute for Health and Care Excellence
Comparator companies Eurocept International (confidentiality agreement not signed, not participating)
  Immedica (confidentiality agreement not signed, not participating)
Evidence review group National Institute for Health Research Health Technology Assessment Programme (NETSCC)
  School of Health and Related Research (ScHARR)
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
  Welsh Government
  Scottish Medicines Consortium
  Welsh Health Specialised Services Committee
Relevant research groups National Institute for Health Research


Key events during the development of the guidance:

Date Update
17 January 2024 Invitation to participate
21 September 2023 Please note that following on from a request received from the company, the timelines for this appraisal have been revised and the appraisal is now anticipated to begin in early-January 2024. These timings are based on a request from the company to reschedule the initial date set by NICE, in order to facilitate a suitably comprehensive and robust submission.
07 September 2023 Awaiting development. Status change linked to Topic Selection Decision being set to Selected
07 September 2023 Topic selection
07 September 2023 Topic selection
07 September 2023 In progress
17 March 2023 (10:00) Scoping workshop
17 January 2023 - 14 February 2023 Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators
17 January 2023 In progress

For further information on our processes and methods, please see our CHTE processes and methods manual