For information, the company have announced that the phase 3 AMETHIST trial of venglustat for the treatment of GM2 gangliosidosis was discontinued based on the absence of positive trends on clinical endpoints and therefore they will no longer be pursuing a Marketing Authorisation Application in this indication at this time. Following this information the appraisal of venglustat for treating gangliosidoses in people 2 years and over [ID6358] has been discontinued.
 
Status Discontinued
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

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Timeline

Key events during the development of the guidance:

Date Update
30 July 2024 Discontinued. For information, the company have announced that the phase 3 AMETHIST trial of venglustat for the treatment of GM2 gangliosidosis was discontinued based on the absence of positive trends on clinical endpoints and therefore they will no longer be pursuing a Marketing Authorisation Application in this indication at this time. Following this information the appraisal of venglustat for treating gangliosidoses in people 2 years and over [ID6358] has been discontinued.
07 May 2024 Please note that following on from advice received from the company, further information regarding the timelines for this appraisal will be available in due course. In the meantime, NICE will continue to monitor the situation and will provide an update as and when the situation changes.
30 August 2023 Awaiting development. Status change linked to Topic Selection Decision being set to Selected

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