Suggested remit: To appraise the clinical and cost effectiveness of vosoritide within its marketing authorisation for treating achondroplasia in people 4 months and over.
Following the decision by the NICE Prioritisation Board to route the appraisal through the Single Technology Appraisal (STA) process, BioMarin has advised that they are no longer pursuing a Marketing Authorisation Application from the Medicines and Healthcare products Regulatory Agency for this indication at this time. NICE cannot produce recommendations for treatments without a marketing authorisation. Therefore, NICE has decided to suspend this appraisal from its current work programme. Prioritisation Board agreed that criteria 1,3 and 4 of the highly specialised technology framework were not met (see NICE-wide topic prioritisation: the manual) and that therefore the treatment should be routed to the technology appraisals programme. As this appraisal has been referred NICE will continue to monitor any development and will update interested parties if the situation changes.
- Status:
- Suspended
- 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:
- 6488
Email enquiries
If you have any queries please email scopingta@nice.org.uk
Timeline
Key events during the development of the guidance:
| Date | Update |
|---|---|
| 20 November 2025 | Suspended. Following the decision by the NICE Prioritisation Board to route the appraisal through the Single Technology Appraisal (STA) process, BioMarin has advised that they are no longer pursuing a Marketing Authorisation Application from the Medicines and Healthcare products Regulatory Agency for this indication at this time. NICE cannot produce recommendations for treatments without a marketing authorisation. Therefore, NICE has decided to suspend this appraisal from its current work programme. Prioritisation Board agreed that criteria 1,3 and 4 of the highly specialised technology framework were not met (see NICE-wide topic prioritisation: the manual) and that therefore the treatment should be routed to the technology appraisals programme. As this appraisal has been referred NICE will continue to monitor any development and will update interested parties if the situation changes. |
| 28 July 2025 | Note - Note added to the project documents |
| 28 July 2025 | Topic selection |
| 16 April 2025 | Referral |
| 14 March 2025 - 11 April 2025 | Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators: ID6488 |
| 14 March 2025 | In progress. Scoping commenced. |
| 12 December 2024 | Referral |
| 30 October 2024 | Note - Note added to the project documents |
| 21 March 2024 | 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