Suggested remit: To appraise the clinical and cost effectiveness of sodium phenylbutyrate–ursodoxicoltaurine within its marketing authorisation for treating amyotrophic lateral sclerosis in adults.
For information, the company have announced that Phase 3 PHOENIX clinical trial did not meet its primary endpoint of reaching statistical significance (p=0.667) as measured by the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) 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 6246

Email enquiries

Timeline

Key events during the development of the guidance:

Date Update
10 April 2024 Suspended. For information, the company have announced that Phase 3 PHOENIX clinical trial did not meet its primary endpoint of reaching statistical significance (p=0.667) as measured by the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) 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.
06 June 2023 - 04 July 2023 Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators: 6246
31 March 2023 In progress. DHSC referral received
31 January 2023 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