Suggested remit: To appraise the clinical and cost effectiveness of fidanacogene elaparvovec within its anticipated marketing authorisation for treating moderately severe to severe haemophilia B.
The company has informed NICE that it will be withdrawing its evidence submission for this appraisal given Fidanacogene elaparvovec cannot be launched until the expiry of uniQure’s European Patent 3,581,650 in the UK. Therefore, we are suspending the appraisal while we consider the next steps.
- 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:
- 4032
Project Team
- Project lead
- Kate Moore
Email enquiries
If you have any queries please email [email protected]
- External Assessment Group:
- BMJ Technology Assessment Group (BMJ-TAG), BMJ
Stakeholders
- Companies sponsors
- Pfizer (fidanacogene elaparvovec)
- Others
- Department of Health and Social Care
- NHS England
- Patient carer groups
- Gene People
- Haemophilia Society
- Professional groups
- Royal College of Physicians
- Comparator companies
- CSL Behring (etranacogene dezaparvovec)
- Novo Nordisk (eptacog alpha, nonacog beta pegol)
- Pfizer (nonacog alfa)
- Swedish Orphan Biovitrum (eftrenonacog alfa)
- General commentators
- All Wales Therapeutics and Toxicology Centre
- British National Formulary
- Cell and Gene Therapy Catapult
- Department of Health - Northern Ireland
- Haemophilia Scotland
- Healthcare Improvement Scotland
- Medicines and Healthcare products Regulatory Agency
- Scottish Medicines Consortium
- Welsh Government
- Welsh Health Specialised Services Committee
Timeline
Key events during the development of the guidance:
| Date | Update |
|---|---|
| 27 January 2025 | Suspended. The company has informed NICE that it will be withdrawing its evidence submission for this appraisal given Fidanacogene elaparvovec cannot be launched until the expiry of uniQure’s European Patent 3,581,650 in the UK. Therefore, we are suspending the appraisal while we consider the next steps. |
| 04 July 2024 | Declaration of interests |
| 06 June 2024 | Committee meeting |
| 25 October 2023 | Invitation to participate |
| 17 May 2023 - 15 June 2023 | Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators: 4032 |
| 17 May 2023 | In progress. Scoping commencing |
| 12 August 2022 | 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