Your Monthly Medicines Roundup for September 2025

Where things were relatively quiet in September 2024, this year, a lot more has been happening with treatments for inherited metabolic disorders entering or being approved in one of the UK nations. Last month’s positive update in England for Idebenone for people with Leber hereditary optic neuropathy has now been extended to Northern Ireland. 

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 took place on the 18th of September.We are currently awaiting a final recommendation, which is expected to be published in October and finalised in November.

Further details can be found by clicking the button below:

Efzimfotase alfa, also known as ALXN1850, for treating hypophosphatasia in people 2 years and over has been identified by NICE in England as a potential new treatment to be assessed. We currently do not have any information on timelines and will update the community as soon as more information is available. Further details can be found on the NICE website:

Further details can be found by clicking the button below:

NICE’s final guidance recommending Idebenone for the treatment of leber hereditary optic neuropathy (LHON)  in people 12 and over was adopted by the Department of Health in Northern Ireland. This means that Idebenone will soon also be available for people with LHON living in Northern Ireland. Further details can be found here:

Further details about the assessment 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 have submitted a joint patient group submission and participated in a PACE meeting. A PACE, Patient and Clinician Engagement meeting, gives patient groups and clinicians a stronger voice in the SMC decision-making process. Mercaptamine for the treatment of cystinosis will be discussed during SMC’s next meeting on the afternoon of Tuesday 7 October. 

Further details can be found by clicking the button below: