Suggested remit: To appraise the clinical and cost effectiveness of fosdenopterin within its marketing authorisation for treating molybdenum cofactor deficiency type A.
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
Process HST Standard
ID number 6264

Provisional Schedule

Committee meeting 05 September 2024
Expected publication 29 January 2025

Project Team

Project lead Celia Mayers

Email enquiries

External Assessment Group Peninsula Technology Assessment Group (PenTAG), University of Exeter


Companies sponsors Sentynl Therapeutics (Fosdenopterin)
Others Birmingham Children’s Hospital NHS Foundation Trust
  Department of Health and Social Care
  NHS England
  Willink Unit, Genetic Medicine, Manchester University Hospitals NHS Foundation Trust
Patient carer groups Gene People
  Metabolic Support UK
Professional groups Royal College of Physicians
Comparator companies None
General commentators All Wales Inherited Metabolic Disease Service
  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 Government
  Welsh Health Specialised Services Committee
Relevant research groups National Institute for Health Research


Key events during the development of the guidance:

Date Update
08 May 2024 After receiving the external assessment report, NICE has assessed the evidence submissions and report to decide how this appraisal will progress. This appraisal will continue as a highly specialised technology and progress to technical engagement before preparation for the committee meeting. To accommodate the addition of technical engagement, the committee meeting will now be held on 5 September 2024.
22 November 2023 Invitation to participate
30 October 2023 Awaiting development. Status change linked to Topic Selection Decision being set to Selected
30 October 2023 Topic selection
30 October 2023 Topic selection
30 October 2023 In progress
26 September 2023 (14:00) Scoping workshop
26 July 2023 - 23 August 2023 Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators: 6264
15 June 2023 In progress. DHSC referral received
20 March 2023 Awaiting development. Status change linked to Topic Selection Decision being set to Selected

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