For information, the company have announced that fordadistrogene movaparvovec for treating Duchenne muscular dystrophy did not meet efficacy thresholds in the primary analysis of the Phase 3 trial (CIFFREO) and therefore they will no longer be pursuing a Marketing Authorisation Application in this indication at this time.
Therefore, NICE has decided to suspend this appraisal from its current work programme.
As this appraisal has been referred, NICE will continue to monitor any development and will update interested parties as and when 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 | 6133 |
Email enquiries
- If you have any queries please email scheduling@nice.org.uk
Timeline
Key events during the development of the guidance:
| Date | Update |
|---|---|
| 31 July 2024 | Suspended. For information, the company have announced that fordadistrogene movaparvovec for treating Duchenne muscular dystrophy did not meet efficacy thresholds in the primary analysis of the Phase 3 trial (CIFFREO) and therefore they will no longer be pursuing a Marketing Authorisation Application in this indication at this time. Therefore, NICE has decided to suspend this appraisal from its current work programme. As this appraisal has been referred, NICE will continue to monitor any development and will update interested parties as and when the situation changes. |
| 23 February 2024 | Please note that following on from advice received from the company this appraisal has been rescheduled to align with latest regulatory expectations. Further information regarding the timelines for this appraisal will be available in due course. |
| 31 July 2023 | Please note that the appraisal has been scheduled into the NICE programme and further details regarding timings and how you can get involved will be available in due course. |
| 16 May 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