As you will be aware the Department for Health & Social Care has asked NICE to carry out an appraisal of Venglustat for treating gangliosidoses in people 2 years and over [ID6358]. For information, the company have announced that trial did not meet its primary endpoint in 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 6358

Project Team

Project lead scopingta@nice.org.uk

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Timeline

Key events during the development of the guidance:

Date Update
25 September 2024 Suspended. As you will be aware the Department for Health & Social Care has asked NICE to carry out an appraisal of Venglustat for treating gangliosidoses in people 2 years and over [ID6358]. For information, the company have announced that trial did not meet its primary endpoint in 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.
25 July 2024 Awaiting development. Status change linked to Topic Selection Decision being set to Selected
25 July 2024 Topic selection

For further information on our processes and methods, please see our CHTE processes and methods manual