After some technical issues last month, we have a double update for you this month, covering the months of August and September 2026. We have two final decisions to share from the Scottish Medicines Consortium, a final HealthTech guidance from NICE and 10 procedural updates, all from NICE. 

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.  

Hydrocortisone for treating people with adrenal insufficiency, which is common in people with X-linked adrenoleukodystrophy, was not assessed by SMC in Scotland in absence of a submission by the company. SMC has not recommended hydrocortisone on this basis; however, this may be revisited if the company does make a submission in the future.

Full details of this decision can be found here: https://scottishmedicines.org.uk/medicines-advice/hydrocortisone-efmody-non-sub-smc2971/  

Leriglitazone for treating cerebral adrenoleukodystrophy in boys and men 2 years and over by NICE in England has restarted. A date for the committee meeting during which the evidence will be discussed has now been set. The meeting will be held on the 18th of February 2027. Families are welcome to observe this meeting virtually and will be able to sign up closer to the date.

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

The assessment of Elamipretide for treating Barth syndrome in people of any age by NICE in England has started. Together with Barth Syndrome UK we have run a survey among the Barth Syndrome community and will be responding with a patient group submission. Currently, the committee meeting, during which the evidence will be discussed, has been set for the 18th of April 2027.  Families are welcome to observe this meeting virtually and will be able to sign up closer to the date.

Further details about the assessment can be found here: https://www.nice.org.uk/guidance/awaiting-development/gid-hst10073  

Venglustat for treating people with Fabry disease was previously identified as a new treatment option for assessment by NICE in England. NICE has shared that timelines for this appraisal will be shared shortly. Once these are available, we will also provide an update on our website.

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

Efzimfotase alfa, also known as ALXN1850, for treating hypophosphatasia in people 2 years and over was identified by NICE in England as a potential new treatment to be assessed in September last year. Timelines were updated in July and have been updated again since. The assessment is currently expecting to commence in April 2027.  

Further details can be found on the NICE website: https://www.nice.org.uk/guidance/awaiting-development/gid-ta11838  

Triheptanoin for treating long-chain fatty acid oxidation disorders (CACT , CPTI, CPTII, LCHAD, TFP, VLCAD ) was identified by NICE in November 2023. The start date of this appraisal has been delayed several times. The latest update indicates that this appraisal is anticipated to begin in early October 2027; which could see triheptanoin be accessible on the NHS in England in 2028. In the meantime, people with an LC-FAOD across the UK can access triheptanoin via an Early Access to Medicines Scheme.

Further details of the appraisal can be found here: https://www.nice.org.uk/guidance/awaiting-development/gid-ta11473 

Further details on early access to triheptanoin can be found here: https://metabolicsupport.org/news-and-events/latest-news/update-on-the-assessment-of-triheptanoin-for-lc-faods/  

The HealthTech guidance on the use of ex-situ machine perfusion devices for the preservation of deceased donor livers for transplantation was published end of August.

Full details can be found here: https://www.nice.org.uk/guidance/awaiting-development/gid-hte10066 

Atidarsagene autotemcel for treating people with metachromatic leukodystrophy has been assessed by SMC in Scotland. SMC has accepted atidarsagene autotemcel for use within NHSScotland.

Full details of this assessment can be found here: https://scottishmedicines.org.uk/medicines-advice/atidarsagene-autotemcel-libmeldy-uo-reassessment-smc2886/ 

We previously reported that Setrusumab for treating osteogenesis imperfecta was identified for assessment by NICE in England. NICE have disclosed that timelines for the assessment will soon be announced. As soon as more information is available we will provide an update.

Further details can be found on the NICE website: https://www.nice.org.uk/guidance/awaiting-development/gid-ta11921 

We previously reported that Nedosiran for treating primary hyperoxaluria type 1 in people 6 years and over was identified for assessment by NICE in England. This assessment has now been suspended because the company is no longer pursuing marketing authorization from the Medicines and Healthcare products Regulatory Agency, the organization who decides whether a medication is safe and can be prescribed in the UK.

Further details can be found here: https://www.nice.org.uk/guidance/awaiting-development/gid-ta11292  

The draft guidance for obicetrapib and obicetrapib–ezetimibe for treating primary hypercholesterolaemia or mixed dyslipidaemia was published by NICE in England in late August. Unfortunately, the initial draft guidance was to not recommend obicetrapib and obicetrapib–ezetimibe. Together with representatives from FH Europe, we have worked on a response, which was submitted by FH Europe in September. A second committee meeting has now been scheduled for the 6th of October. During next month’s medicine round up, we expect to be able to provide an update on this meeting, as well as expected timelines for the final publication. 

Further details can be found on the NICE website: https://www.nice.org.uk/guidance/indevelopment/gid-ta11649  

In parallel, NICE have also identified lerodalcibep as a new treatment for assessment for treating people with hypercholesterolaemia, including familial hypercholesterolaemia. 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-ta12541  

In our last update in July 2026, we shared that the assessment of Doxecitine–doxribtimine for treating people with thymidine kinase 2 deficiency at any age remained paused in England. Late September, NICE shared that the assessment is expected to resume mid-December 2026. Together with the Lily Foundation, we will work on this assessment. 

Further details can be found here:  https://www.nice.org.uk/guidance/indevelopment/gid-hst10063