This month we have four updates for you about the (upcoming) assessment of various new medications in England.

If you find yourself wanting explanations of the updates or needing a listening ear, you can reach out to us via 0800 652 3181 or email us at contact@metabolicsupport.org.  

Venglustat for treating people with Fabry disease was identified as a new treatment option for assessment by NICE in England. Currently, timelines for this appraisal are not yet known. 

Further details can be found here: https://www.nice.org.uk/guidance/awaiting-development/gid-ta12452

Last month, we provided the update that plozasiran for treating people with familial chylomicronaemia syndrome (FCS) changed its process through NICE in England. This month, it was announced that olezarsen will be following a similar pathway.  

As communicated last month, most often, NICE assesses new medicines based on their costs and effectiveness (how much more needs to be paid for better health outcomes?). In this case, the new medicine will only be assessed as a cost-comparison. This generally means that there is an expectation that the new medicine will be just as effective as the existing standard of care (in this case volanesorsen and dietary restrictions) and thus only a comparison on costs will be made (which goes beyond treatment costs; it also includes for example the number of expected appointments with healthcare providers and how these are impacted by the different medication options).  

Based on this revised pathway, the committee meeting scheduled for end of June was cancelled and a draft decision on olezarsen  is expected by 26 August 2026. 

Further details for olezarsen can be found here: https://www.nice.org.uk/guidance/indevelopment/gid-ta11678 

We have previously given updates about primary hypercholesterolaemia or mixed dyslipidaemia in the context of the ongoing appraisal of obicetrapib and obicetrapib–ezetimibe by NICE in England. In the meantime, an update on the already approved bempedoic acid with ezetimibe has been issued by NICE. This update is in relation to a historical commercial arrangement and the pricing of bempedoic acid with ezetimibe. This should not affect established access.  

Further details can be found on the NICE website: https://www.nice.org.uk/guidance/ta694 

In parallel, NICE have also identified laroprovstat as a new treatment for assessment for treating people with primary hypercholesterolaemia or mixed dyslipidaemia disease. No timelines for this assessment are currently available.  

Further details can be found on the NICE website: https://www.nice.org.uk/guidance/awaiting-development/gid-ta12477