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

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