Your Monthly Medicines Roundup for April 2025

Metabolic Support UK have six updates relating to treatments for inherited metabolic disorders to share for the month of March. One medication received its final recommendation, one medication moved to the scoping stage, one medication will soon have its second committee meeting, one medication had its Managed Access Agreement extended, and two new treatments have been added to NICE’s workstream. 

You can find details below: 

Pegzilarginase for treating arginase-1 deficiency is currently being accessed by NICE in England under their highly specialised technology pathway. The second committee meeting is expected to happen on the 22nd of May. 

For further details about the assessment, click the first button. A registration link to attend the committee meeting as an observer will be available via the second button in due course (under the HSTEC):

Elamipretide for treating Barth syndrome in people of any age 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.

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

Neuronal Ceroid Lipofuscinosis-Type 2 (batten disease type 2) – Last month, we let you know that a committee meeting for Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2 by NICE in England was expected to take place in April. This meeting has taken place. We are now awaiting an update. In the meantime, NICE, NHS England and BioMarin have announced that the Managed Access Agreement has been extended for a duration of three months.

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

NICE in England have announced that they will be assessing a new technique for liver transplants: machine perfusion devices. As numerous of our community members have or will undergo a liver transplant in the course of their life, we will keep tabs on this appraisal and may reach out to you to hear about your experiences.

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

As reported in our February update, NICE in England have released the final recommendation for Olipudase alfa in the treatment of people living with Niemann-Pick type AB and B. This decision was officially published at the beginning of April. Unfortunately, NICE do not recommend Olipudase alfa. They reached this conclusion based on the evidence presented by the Company. NICE stated that the evidence shows that, after one year of treatment, lung function is improved and the size of the spleen is reduced. These improvements become more gradual as the condition stabilises. This means that the treatment shows a positive effect in managing the condition. Unfortunately, the cost associated with the treatment are considered too high for NICE to recommend this as acceptable use of NHS resources. For further support, we recommend reaching out to Niemann-Pick UK.

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

Scoping for doxecitine–doxribtimine for treating thymidine kinase 2 deficiency in people was initiated by NICE In England. Metabolic Support UK, together with the Lily Foundation will be responding to the scoping stage.

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