Suggested remit: To evaluate the benefits and costs of arimoclomol within its marketing authorisation for treating Niemann-Pick disease type C for national commissioning by NHS England.
On 23 February 2022, the company (Orphazyme) issued a statement that it is currently assessing its strategic options following feedback on arimoclomol from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP). Therefore, this evaluation will be suspended. NICE will continue to monitor any developments and will update stakeholders if the situation changes.
 
Status Suspended
Decision Selected
Process HST
ID number 1312

Project Team

Project lead Gavin Kenny

Email enquiries

Stakeholders

Companies sponsors • Orphazyme (arimoclomol)
Others • Addenbrooke's Lysosomal Disorders Unit
  • Birmingham Children’s Hospital NHS Foundation Trust Lysosomal Storage Disorders Unit
  • Department of Health and Social Care
  • Great Ormond Street Hospital Metabolic Unit
  • NHS England
  • Royal Free Lysosomal Storage Disorders Unit
  • Royal Manchester Children’s Hospital
  • Salford Royal NHS Foundation Trust Mark Holland Metabolic Unit
  • University College London Hospitals NHS Foundation Trust Lysosomal Disorders Unit
  • Willink Unit, Genetic Medicine, Central Manchester Foundation Trust
Patient carer groups • Brain Charity
  • Contact
  • Findacure
  • Genetic Alliance UK
  • Gene People
  • Metabolic Support UK
  • Neurological Alliance
  • Niemann-Pick UK
  • South Asian Health Foundation
  • Specialised Healthcare Alliance
  • UK LSD Patient Collaborative Group
Professional groups • Association of British Neurologists
  • Association of Genetic Nurses and Counsellors
  • British Association for the Study of the Liver
  • British Association of Occupational Therapists and College of Occupational Therapists
  • British Inherited Metabolic Disease Group
  • British Neuropathological Society
  • British Paediatric Neurology Association
  • British Society for Gene and Cell Therapy
  • British Society for Genetic Medicine
  • Institute of Neurology
  • National Metabolic Biochemistry Network
  • Neonatal and Paediatric Pharmacists Group
  • 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
  • UK Genetic Testing Network
  • UK Health Forum
Associated public health groups • Public Health Wales
  • UK Health Security Agency
Comparator companies • Accord Healthcare (miglustat)
  • Janssen (miglustat)
  • Piramal Critical Care (miglustat
General commentators • All Wales Therapeutics and Toxicology Centre
  • Allied Health Professionals Federation
  • Board of Community Health Councils in Wales
  • British National Formulary
  • Care Quality Commission
  • Cell and Gene Therapy Catapult
  • Department of Health, Social Services and Public Safety for Northern Ireland
  • Healthcare Improvement Scotland
  • Inherited Metabolic and Lysosomal Disease Service, Cardiff and Vale UHB
  • Medicines and Healthcare products Regulatory Agency
  • National Association of Primary Care
  • National Pharmacy Association
  • National Services Division
  • NHS Alliance
  • NHS Confederation
  • Scottish Medicines Consortium
  • Welsh Government
  • Welsh Health Specialised Services Committee
Relevant research groups • Brain Research UK
  • Cochrane Hepato-Biliary Group
  • Cochrane Metabolic & Endocrine Disorders Group
  • Cochrane UK
  • Genomics England
  • MRC Clinical Trials Unit
  • National Institute for Health Research

Timeline

Key events during the development of the guidance:

Date Update
01 March 2022 Suspended. On 23 February 2022, the company (Orphazyme) issued a statement that it is currently assessing its strategic options following feedback on arimoclomol from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP). Therefore, this evaluation will be suspended. NICE will continue to monitor any developments and will update stakeholders if the situation changes.
17 December 2021 Invitation to participate
17 May 2021 Please note that following on from advice received from the company this evaluation has been rescheduled. Therefore, we now anticipate that the evaluation will begin during early November 2021 when we will write to you about how you can get involved. The deadline for submissions is expected in approximately mid-Jan 2022.
09 October 2020 (14:00) Scoping workshop
19 August 2020 - 17 September 2020 Consultation on suggested remit, draft scope and provisional matrix of consultees and commentators

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