The manufacturer of idebenone has informed NICE that they have “withdrawn the European marketing authorization application and ended the global development program for Puldysa [idebenone].”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.
| Status | Suspended |
| Process | STA 2018 |
| ID number | 1092 |
Project Team
| Project lead | Jeremy Powell |
Email enquiries
- If you have any queries please email TACommB@nice.org.uk
| Evidence Review Group / Assessment Group | Kleijnen Systematic Reviews Ltd |
Consultees
| 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 |
Commentators
| 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 |
|---|---|
| 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 how we develop guidance, please see our page about NICE technology appraisal guidance