Your Monthly Medicines Roundup for August 2025

August has been a busy month for metabolic medicine announcements. There are updates to 9 medicines and one medical device regarding 9 different disorders across England and Scotland. This includes a positive update for Idebenone for the treatment of Leber hereditary optic neuropathy in people 12 and over. 

You can find details below: 

Pegzilarginase for treating arginase-1 deficiency is currently being assessed by NICE in England under their highly specialised technology pathway. The third evaluation committee meeting has now been scheduled for the 18th of September.  

Further details can be found by clicking the button below:

Scoping was completed for Plozasiran and Olezarsen, two medicines which each treat familial chylomicronaemia syndrome, by NICE in England, this ran until 20 August 2025. Once more details about the process have been announced we will share these. Further details for plozasiran can be found here:

Further details for Plozasiran and Olezarsen can be found by clicking the buttons below:

Final guidance recommending Idebenone for the treatment of leber hereditary optic neuropathy in people 12 and over by NICE in England was published on the 28th of August. The cost-effectiveness estimates for Idebenone are within the range that NICE normally considers an acceptable use of NHS resources and therefore it was recommended for this indication.

Further details about the assessment can be found by clicking the button below:

This month NICE in England completed a consultation of the scoping documents for a new technique for liver transplants: machine perfusion devices. This scoping consultation allowed patient and medical experts to ensure the right metrics are evaluated when NICE appraises the cost effectiveness of this medical technology.

Further details can be found by clicking the button below:

As per our previous updates, the assessment of triheptanoin for the treatment of people with one of the long-chain fatty acid oxidation disorders (CPTI, CACT, CPTII, VLCAD, TFP and LCHAD) is currently ongoing. The company has requested an additional delay in the appraisal process to allow them to provide a suitably comprehensive and robust submission to NICE in England, the appraisal is now expected to begin in September 2026.

Further details can be found by clicking the button below:

The assessment of mercaptamine for the treatment of nephropathic cystinosis is currently undergoing a resubmission process by the Scottish Medicines Consortium in Scotland. Metabolic Support UK, Cystinosis Foundation UK and Kidney Research UK were invited by SMC to provide a patient group submission which was completed in early August. The SMC will now discuss the evidence at the New Drug Committee and will make a decision, together with other stakeholders, on whether a PACE meeting will need to be held. A PACE, Patient and Clinician Engagement meeting, gives patient groups and clinicians a stronger voice in the SMC decision-making process. 

Further details can be found by clicking the button below:

Doxecitine–doxribtimine for treating people with thymidine kinase 2 deficiency at any age will now be assessed through the highly specialised technology pathway by NICE in England. Metabolic Support UK, together with The Lily Foundation, have been invited to provide a patient group submission, which we are currently working on before the first committee meeting occurs in March next year. Further details can be found here:

Further details can be found by clicking the button below:

In August, unfortunately, the final draft guidance not recommending Cerliponase alfa for the treatment batten disease type 2 was published by NICE in England. This guidance is expected to be officially published on the 17th of September. 

Further details can be found by clicking the button below:

Dersimelagon for treating erythropoietic protoporphyria and X-linked protoporphyria in people 12 years and over. On the 26th of August NICE confirmed that in England, timelines for the appraisal of this medicine are being confirmed as the pharmaceutical company developing this medicine are finalising their regulatory filing plans with the Medicines and Healthcare products Regulatory Agency (who ensure that new medicines meet appropriate standards of safety, quality, and effectiveness).

Further details can be found by clicking the button below: