Following on from information provided to NICE by the company in May 2018 the appraisal of Eteplirsen for treating Duchenne muscular dystrophy [ID1003] 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 | HST |
| ID number | 1003 |
Email enquiries
- If you have any queries please email scheduling@nice.org.uk
Stakeholders
| Companies sponsors | Sarepta Therapeutics |
| Others | Department of Health and Social Care |
| NHS England | |
| Patient carer groups | Action Duchenne |
| Action for Sick Children | |
| Alex’s Wish | |
| Arthritis & Musculoskeletal Alliance | |
| Children’s Society | |
| Contact a Family | |
| Disability Rights UK | |
| DMD Pathfinders | |
| Duchenne Family Support Group | |
| Duchenne Now | |
| Duchenne UK | |
| Findacure | |
| Genetic Alliance | |
| Harrison’s Fund | |
| Information Advice and Support Service Network | |
| Joining Jack | |
| 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 |
| 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 Heart Rhythm Society | |
| British Institute of Musculoskeletal Medicine | |
| British Orthopaedic Association | |
| British Paediatric Neurology Association | |
| British Paediatric Respiratory Society | |
| British Society for Genetic Medicine | |
| British Society of Rehabilitation Medicine | |
| British Thoracic Society | |
| Chartered Society of Physiotherapy | |
| Physiotherapy Pain Association | |
| Primary Care Respiratory Society | |
| Royal College of Anaesthetists | |
| 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 | |
| The National Congenital Anomaly and Rare Disease Registration Service | |
| UK Genetic Testing Network | |
| UK Clinical Pharmacy Association | |
| 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 | |
| NHS National Services Scotland | |
| Scottish Medicines Consortium | |
| Welsh Government | |
| Welsh Health Specialised Services Committee | |
| Relevant research groups | British Myology Society |
| Cochrane Cystic Fibrosis and Genetic Disorders Group | |
| Cochrane Musculoskeletal Group | |
| Duchenne Research Fund | |
| MRC Centre for Neuromuscular Diseases | |
| MRC Clinical Trials Unit | |
| Newcastle University John Walton Centre for Muscular Dystrophy Research | |
| North Star Clinical Network | |
| National Institute for Health Research | |
| TREAT-NMD |
Timeline
Key events during the development of the guidance:
| Date | Update |
|---|---|
| 15 November 2022 | Discontinued. Following on from information provided to NICE by the company in May 2018 the appraisal of Eteplirsen for treating Duchenne muscular dystrophy [ID1003] was suspended from NICE’s work programme. As no further information has been received from the company the topic has been discontinued. |
| 04 July 2018 | Suspended. Suspended |
| 04 July 2018 | As you will be aware, the Department for Health and Social Care has asked NICE to carry out an evaluation of eteplirsen for treating Duchenne muscular dystrophy. However, the company have advised that during June 2018 the Committee for Medicinal Products for Human Use (CHMP) adopted a negative opinion, recommending the refusal of the marketing authorisation for eteplirsen for treating Duchenne muscular dystrophy. The company have already requested a data re-examination by the CHMP, the outcome of this will be known during the Autumn. Therefore, as is usual practice with a negative CHMP opinion, NICE has decided to suspend this appraisal on its work programme but will continue to monitor the regulatory situation for eteplirsen and will update interested parties if the situation changes. |
| 11 May 2018 | Please note that following advice received from the company around a delay to regulatory timings. The deadline for submissions is expected to be delayed. Consequently the forthcoming committee meeting discussion scheduled for 23 August 2018 will also be delayed. The revised committee meeting discussion date is to be confirmed. |
| 16 March 2018 | Invitation to participate |
| 13 March 2018 | In progress. Topic referred |
| 01 March 2017 | Referral |
| 04 August 2016 - 25 August 2016 | 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