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 04 July 2024
Expected publication 11 September 2024

Project Team

Project lead Celia Mayers

Email enquiries


Companies sponsors Sentynl Therapeutics (Fosdenopterin)
Others Birmingham Children’s Hospital NHS Foundation Trust
  Bradford Royal Infirmary
  Bristol Royal Hospital for Children, Southwest Regional Metabolic Department
  Department of Health and Social Care
  Evelina London Children’s Hospital, Guy’s and St Thomas’ NHS Foundation Trust
  Great Ormond Street Hospital Metabolic Unit
  NHS England
  Nottingham Children’s Metabolic Service, Nottingham University Hospitals NHS Trust
  Sheffield Children’s NHS Foundation Trust
  University Hospitals of Leicester NHS Trust
  University Hospital Southampton NHS Foundation Trust
  Willink Unit, Genetic Medicine, Manchester University Hospitals NHS Foundation Trust
Patient carer groups Anthony Nolan
  Brain and Spine Foundation
  Brain Charity
  Disability Rights UK
  Epilepsy Action
  Epilepsy Society
  Gene People
  Genetic Alliance UK
  Metabolic Support UK
  National Council of Palliative Care
  Neurological Alliance
  Sue Ryder
  South Asian Health Foundation
  Specialised Healthcare Alliance
  Together for Short Lives
  Young Epilepsy
Professional groups Association of British Neurologists
  Association of Genetic Nurses & Counsellors
  British Inherited Metabolic Disease Group
  British Neuropathological Society
  British Paediatric Epilepsy Group
  British Society for Genetic Medicine
  Epilepsy Nurses Association
  International League Against Epilepsy UK Chapter
  Institute of Neurology
  National Neuroscience Advisory Group
  National Metabolic Biochemistry Network
  Neonatal and Paediatric Pharmacists Group
  Primary Care and Community Neurology Society
  Royal College of General Practitioners
  Royal College of Nursing
  Royal College of Paediatrics & Child Health
  Royal College of Pathologists
  Royal College of Physicians
  Royal Pharmaceutical Society
  Royal Society of Medicine
  UK Clinical Pharmacy Association
Associated public health groups Public Health Wales
  UK Health Security Agency
Comparator companies None
General commentators All Wales Inherited Metabolic Disease Service
  All Wales Therapeutics and Toxicology Centre
  Allied Health Professionals Federation
  Board of Community Health Councils in Wales
  British National Formulary
  Care Quality Commission
  Department of Health, Social Services and Public Safety for Northern Ireland
  Healthcare Improvement Scotland
  Medicines and Healthcare products Regulatory Agency
  National Association of Primary Care
  National Pharmacy Association
  National Services Division
  Neurological Alliance of Scotland
  NHS Confederation
  Scottish Medicines Consortium
  Wales Neurological Alliance
  Welsh Government
  Welsh Health Specialised Services Committee
Relevant research groups Brain Research UK
  Cochrane Metabolic & Endocrine Disorders Group
  Cochrane UK
  Genomics England
  MRC Clinical Trials Unit
  National Hospital for Neurology and Neurosurgery
  National Institute for Health Research


Key events during the development of the guidance:

Date Update
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