Suggested remit: To appraise the clinical and cost effectiveness of intrathecal onasemnogene abeparvovec within its marketing authorisation for treating spinal muscular atrophy.
- 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:
- 6556
Provisional Schedule
- Committee meeting: 1:
- 07 July 2026
- Expected publication:
- 26 August 2026
- Expected publication:
- 28 October 2026
Project Team
- Project lead
- Leena Issa
Email enquiries
If you have any queries please email TACommC@nice.org.uk
- External Assessment Group:
- BMJ Technology Assessment Group (BMJ-TAG), BMJ
Stakeholders
- Companies sponsors
- Novartis Pharmaceuticals UK (onasemnogene abeparvovec)
- Others
- Department of Health and Social Care
- NHS England
- Sheffield Children’s NHS Foundation Trust
- Patient carer groups
- Muscular Dystrophy UK
- Spinal Muscular Atrophy UK
- TreatSMA
- Professional groups
- Adult SMA Reach
- Association of British Neurologists
- Neonatal and Paediatric Pharmacists Group
- Royal College of Physicians
- SMA Reach UK
- Treat-NMD
- Associated public health groups
- None
- Comparator companies
- Biogen (nusinersen) (confidentiality agreement signed, participating)
- Roche (risdiplam) (confidentiality agreement signed, participating)
- Novartis Pharmaceuticals UK (onasemnogene abeparvovec [intravenous infusion] (confidentiality agreement not signed, not participating)
- General commentators
- All Wales Therapeutics and Toxicology Centre
- British National Formulary
- Department of Health - Northern Ireland
- Healthcare Improvement Scotland
- Medicines and Healthcare products Regulatory Agency
- NHS Wales Joint Commissioning Committee
- Scottish Medicines Consortium
- Welsh Government
- Relevant research groups
- John Walton Muscular Dystrophy Research Centre
Timeline
Key events during the development of the guidance:
| Date | Update |
|---|---|
| 24 November 2025 | Invitation to participate |
| 02 October 2025 | Note - Note added to the project documents |
| 04 August 2025 - 02 September 2025 | Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators: 6556 |
| 04 August 2025 | In progress. Scoping commencing |
| 08 April 2025 | 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