This month we have four updates for you about the (upcoming) assessment of various new medications; one from Scotland, and three from 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.  

Velmanase alfa as an enzyme replacement therapy for the treatment of non-neurological manifestations in patients with mild to moderate alpha-mannosidosis is expected to be reassessed by SMC in Scotland this year, under its ultra-orphan pathway. In the last assessment in 2022, SMC did not recommend Velmanase for people with alpha-mannosidosis. Further details can be found here:

Further details about the assessment can be found here: https://scottishmedicines.org.uk/medicines-advice/velmanase-alfa-lamzede-ultra-orphan-pathway-reassessment-smc2952/

As indicated last month, the assessment of Leriglitazone for treating cerebral adrenoleukodystrophy in boys and men 2 years and over by NICE in England has restarted. All stakeholders (the company marketing the drug, medical centres, patient groups, professional groups, and others) have now been invited to submit their evidence and suggested experts.

Further details about the assessment can be found here: https://www.nice.org.uk/guidance/indevelopment/gid-ta11445 

We have previously updated you about the progress that plozasiran, a medicine which can treat familial chylomicronaemia syndrome (FCS), is making progress through the NICE process in England.

This month, it was announced that the assessment would continue in a different format. 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 23 July 2026 is now cancelled and a draft decision on plozasiran is expected by 17 September 2026.

Further details for plozasiran can be found here: https://www.nice.org.uk/guidance/indevelopment/gid-ta11793

Olezarsen, another medicine which can treat FCS, is also going through the NICE process in England. The committee meeting for Olezarsen will be held on the afternoon of the 25 June 2026. People from the community who are interested in attending can register their interest via the following link: https://meetnice.zoom.us/meeting/register/e3QnbZJ_RK-TP6PCpXVCRA#/registration

Over the past two months we shared updates around the assessment of rebisufligene etisparvovec for treating mucopolysaccharidosis type IIIA, which was initiated by NICE in England in March. In April, the scoping workshop took place. This month, NICE have shared that the timelines for this assessment have been delayed to ensure it aligns with regulatory approval (i.e. when the MHRA, who decides whether a medication is safe in humans, approves the medicine). 

Further details about this assessment can be found here: https://www.nice.org.uk/guidance/awaiting-development/gid-ta11701