Your Monthly Medicines Roundup for July 2025
Your Monthly Medicines Roundup for July 2025
This month we have six updated, regarding five different disorders across both England and Scotland.
You can find details below:
Classical galactosemia
The assessment of Govorestat for treating classic galactosemia in people 2 to 65 years has been suspended by NICE in England because the company are no longer pursuing marketing authorization from the regulator in the UK, the Medicines and Healthcare products Regulatory Agency. Without regulatory approval, a medicine cannot be marketed in the UK. The company may still pursue this in the future and we will keep you updated if and when things evolve.
Further details can be found by clicking the button below:
Familial chylomicronaemia syndrome
Scoping was initiated for Plozasiran and Olezarsen, two medicines which each treat familial chylomicronaemia syndrome, by NICE in England, and will run until 20 August 2025. Once more details about the process have been announced we will share these.
Further details for Plozasiran and Olezarsen can be found by clicking the buttons below:
Liver transplant
In July, NICE in England held a scoping workshop around the assessment of a new technique for liver transplants: machine perfusion devices. During this workshop it became clear that this technique is already in use in all UK transplant centres. The assessment will now proceed to routine use assessment.
Further details can be found by clicking the button below:
Nephropathic Cystinosis
The assessment of mercaptamine for the treatment of nephropathic cystinosis is currently ongoing by the Scottish Medicines Consortium in Scotland. Metabolic Support UK, Cystinosis Foundation UK and Kidney Research UK have been invited by SMC to provide a patient group submission which will be completed early August. After this, SMC will 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:
Neuronal Ceroid Lipofuscinosis-Type 2 (batten disease type 2)
In July, the fourth committee meeting for batten disease type 2 was held by NICE in England. Comments from the consultation that ran during May and June were discussed. Currently, NICE expect to publish their final guidance for Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2 by 17 September 2025
Further details can be found by clicking the button below:
