To appraise the clinical and cost effectiveness of idebenone within its marketing authorisation for treating Duchenne muscular dystrophy.

Following on from information provided to NICE by the company in October 2020 the appraisal of Idebenone for treating Duchenne muscular dystrophy [ID1092] was suspended from NICE’s work programme. As no further information has been received from the company the topic has been discontinued.

Status:
Discontinued
Technology type:
Medicine
Decision:
Selected
Process:
TA
ID number:
1092

Email enquiries

If you have any queries please email scheduling@nice.org.uk


External Assessment Group:
Kleijnen Systematic Reviews Ltd

Stakeholders

Companies sponsors
Santhera Pharmaceuticals (idebenone)
Others
Department of Health
 
NHS England
 
NHS South Cheshire CCG
 
NHS West London CCG
 
Welsh Government
Patient carer groups
Action on Pain
 
Action Duchenne
 
Action for Sick Children
 
Alex's Wish
 
Arthritis & Musculoskeletal Alliance
 
Contact a Family
 
Disability Rights UK
 
Duchenne Family Support Group
 
Duchenne Now
 
Duchenne Research Fund
 
Duchenne UK
 
Genetic Alliance UK
 
Harrison’s Fund
 
Information Advice and Support Service Network
 
Leonard Cheshire Disability
 
Muscular Dystrophy UK
 
Muslim Council of Britain
 
National Children’s Bureau
 
South Asian Health Foundation
 
Specialised Healthcare Alliance
 
Together for Short Lives
Professional groups
Association of Anaesthetists of Great Britain & Ireland
 
Association of British Neurologists
 
Association of Genetic Nurses & Counsellors
 
Association of Respiratory Nurse Specialists
 
Association of Surgeons of Great Britain and Ireland
 
British Cardiovascular Society
 
British Dietetic Association
 
British Institute of Musculoskeletal Medicine
 
British Orthopaedic Association
 
British Paediatric Neurology Association
 
British Paediatric Respiratory Society
 
British Society for Children's Orthopaedic Surgery
 
British Society for Gene and Cell Therapy
 
British Society for Genetic Medicine
 
British Society for Human Genetics
 
British Society of Rehabilitation Medicine
 
British Thoracic Society
 
Chartered Society of Physiotherapy
 
Primary Care Respiratory Society UK
 
Royal College of General Practitioners
 
Royal College of Nursing
 
Royal College of Paediatrics and Child Health
 
Royal College of Pathologists
 
Royal College of Physicians
 
Royal College of Surgeons
 
Royal Pharmaceutical Society
 
Royal Society of Medicine
 
UK Genetic Testing Network
 
UK Clinical Pharmacy Association
 
The National Congenital Anomaly and Rare Disease Registration Service
 
The North Star Project
Associated public health groups
Public Health England
 
Public Health Wales
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
 
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
 
NHS Alliance
 
NHS Commercial Medicines Unit
 
NHS Confederation
 
Scottish Medicines Consortium
 
National Services division for Scotland
 
Welsh Health Specialised Services Committee
Relevant research groups
British Myology Society
 
Cochrane Cystic Fibrosis and Genetic Disorders Group
 
Cochrane Musculoskeletal Group
 
John Walton Centre for Muscular Dystrophy (Newcastle University)
 
MRC Centre for Neuromuscular Diseases
 
MRC Clinical Trials Unit
 
National Institute for Health Research
 
TREAT-NMD

Timeline

Key events during the development of the guidance:

Date Update
21 December 2022 Discontinued. Following on from information provided to NICE by the company in October 2020 the appraisal of Idebenone for treating Duchenne muscular dystrophy [ID1092] was suspended from NICE’s work programme. As no further information has been received from the company the topic has been discontinued.
22 October 2020 Suspended. The manufacturer of idebenone has informed NICE that they have “withdrawn the European marketing authorization application and ended the global development program for Puldysa [idebenone].” More information is available on the company’s website: http://www.santhera.com/assets/files/press-releases/2020-10-06_SiderosPuldysa_e_final.pdf NICE is therefore stopping work on this appraisal and will ask for it to be removed from our work programme.
02 July 2020 Invitation to participate
21 April 2020 Topic update: this appraisal has not been defined as therapeutically critical (please follow the link titled rapid guidelines and evidence summaries within the yellow banner at the top of the page for information on recent changes to the way we work). The appraisal will therefore be paused. At this stage, we are unable to provide alternative timelines as to when the appraisal may be re-started but we will provide updates as and when this information becomes clear.
18 March 2020 As you will be aware, the Department for Health and Social Care has asked NICE to carry out a Single Technology Appraisal of Puldysa (idebenone). Please note that following on from advice received from the company this appraisal has been rescheduled. Therefore, we now anticipate that the appraisal will begin after the summer of 2020.
20 November 2019 - 18 December 2019 Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators
22 November 2019 In progress. In progress
22 February 2018 Suspended. Suspended
10 March 2017 (10:00) Scoping workshop (London)
19 January 2017 - 16 February 2017 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