Suggested remit: To appraise the clinical and cost effectiveness of givinostat within its marketing authorisation for treating Duchenne muscular dystrophy in people 6 years and over.
- Status:
- In progress
- Technology type:
- Medicine
- Decision:
- Selected
- Reason for decision:
- Anticipate the topic will be of importance to patients, carers, professionals, commissioners and the health of the public to ensure clinical benefit is realised, inequalities in use addressed, and help them make the best use of NHS resources
- Process:
- STA Standard
- ID number:
- 6323
Project Team
- Project lead
- Jennifer Upton
Email enquiries
If you have any queries please email TACommA@nice.org.uk
Stakeholders
- Companies sponsors
- ITF Pharma UK (givinostat)
- Others
- Department of Health and Social Care
- NHS England
- The John Walton Muscular Dystrophy Research Centre, Newcastle
- Patient carer groups
- Action Duchenne
- Duchenne UK
- Gene People
- Muscular Dystrophy UK
- Professional groups
- Association of British Neurologists
- British Paediatric Neurology Association
- British Society for Paediatric Endocrinology and Diabetes
- British Society of Physical and Rehabilitation Medicine
- Chartered Society of Physiotherapy
- Royal College of Physicians
- Associated public health groups
- None
- Comparator companies
- PTC Therapeutics (ataluren)
- General commentators
- All Wales Therapeutics and Toxicology Centre
- Board of Community Health Councils in Wales
- British National Formulary
- Department of Health - Northern Ireland
- Healthcare Improvement Scotland
- Medicines and Healthcare products Regulatory Agency
- Scottish Medicines Consortium
- Welsh Government
- NHS Wales Joint Commissioning Committee
- Relevant research groups
- None
Timeline
Key events during the development of the guidance:
| Date | Update |
|---|---|
| 23 October 2025 | Committee meeting: 2 |
| 12 August 2025 - 12 September 2025 | Call for evidence |
| 18 August 2025 | Following its July meeting, the committee was unable to reach a full conclusion on givinostat because more information is needed. Consequently, NICE will not be issuing draft guidance for consultation at this stage. The committee has identified specific areas where additional evidence is required to support a fully informed decision. As a result, NICE has launched a four-week targeted call for evidence from stakeholders, ahead of a further committee meeting in October. |
| 10 July 2025 | Committee meeting: 1 |
| 10 July 2025 | Declaration of interests |
| 17 March 2025 | "After receiving the external assessment report, NICE has assessed the evidence submissions and report to decide how this appraisal will progress. This appraisal will continue as a single technology appraisal and progress to technical engagement before preparation for the committee meeting in July 2025. Due to operational issues at NICE although the topic remains a single technology appraisal (STA) it will be discussed by the highly specialised technologies (HST) committee in July. |
| 07 August 2024 | Topic routing was discussed at the NICE Prioritisation Board in June 2024. The Board concluded that the topic was suitable for a Technology Appraisal. Please see project documents for further details. |
| 05 August 2024 | Please note that following on from a request received from the company, the timelines for this appraisal have been revised. The deadline for submissions is expected in approximately early December 2024. These timings are based on a request from the company to reschedule the initial date set by NICE, in order to facilitate a suitably comprehensive and robust submission. |
| 13 June 2024 | Invitation to participate |
| 09 April 2024 - 08 May 2024 | Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators: 6323 |
| 09 April 2024 | In progress. Scoping commenced. |
| 13 July 2023 | Awaiting development. Status change linked to Topic Selection Decision being set to Selected |
For further information on our processes and methods, please see our CHTE processes and methods manual