Your Monthly Medicines Roundup for June 2025

It has been a busy month in terms of public announcements of new access developments for metabolic medicines across the majority of the devolved nations. This month, we will be sharing updates from England, Scotland and Wales. 

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 The second draft guidance was published earlier this month, not recommending pegzilarginase for the inclusion on the NHS. Further details can be found on the NICE website and in our recent news update:

Further details can be found on the NICE website and in our recent news update:

The Scottish Medicines Consortium (SMC) in Scotland recorded a non-submission for pegzilarginase for treating arginase-1 deficiency. In the absence of a submission from the company, pegzilarginase is not recommended for use within NHSScotland. If the company decide to make a submission in the future, SMC will revisit their decision.

Further details can be found via the button below:

Plozasiran for treating people with familial chylomicronaemia syndrome was selected for assessment by NICE In England. Timelines for the next steps of this assessment are currently unclear. We will provide updates as we receive them.

Further details can be found here:

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 a delay in the appraisal process to allow them to align the NICE process in England with the regulatory process at 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 here:

Mercaptamine for the treatment of proven nephropathic cystinosis is currently being considered by the Scottish Medicines Consortium in Scotland. Once we have more information around timelines we will publish an update on our medicines consultation page.

For further details about the assessment, click the button below:

As was shared in our recent news update, the company behind fosdenopterin unfortunately decided to withdraw their application to NICE in England. As a result, fosdenopterin will not be made available on the NHS in England. This decision was also adopted by the AWMSG in Wales, who have excluded fosdenopterin based on the NICE appraisal.

Further details can be found here and in our recent news update: