Your Monthly Medicines Roundup for October 2025

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 NICE have shared is delayed. We hope a final recommendation will be published late November of early December.

Further details can be found by clicking the button below:

PXT3003 for treating Charcot-Marie-Tooth disease type 1A in people aged 16 and over has been awaiting development by NICE in England since April 2023. NICE have included an update on the remit. We are currently enquiring with NICE what this update means in terms of the progression of this assessment.

Further details can be found by clicking the button below:

Plozasiran and Olezarsen, two medicines which each treat familial chylomicronaemia syndrome (FCS) are currently in the process of being assessed by NICE in England. We are working with Action FCS throughout this process and have put together a survey to collect people’s experiences.

If you would like to share your experiences, please share these by clicking the button below. For further information on plozasiran and olezarsen, click the corresponding buttons.

On the 7th of October, the resubmission of mercaptamine for the treatment of nephropathic cystinosis was discussed during the Scottish Medicines Consortium’s committee meeting. Metabolic Support UK, Cystinosis Foundation UK and Kidney Research UK attended and shared the experiences and perspectives of people living with cystinosis and their families. The final outcome of this assessment is due to be published by SMC on the 10th of October.

Further details can be found by clicking the button below:

In August, the final draft guidance not recommending Cerliponase alfa for the treatment batten disease type 2 was published by NICE in England. Both the Batten Disease Family Association and BioMarin, the pharmaceutical company behind the medicine, have appealed NICE’s decision. The appeals were officially logged on the NICE website in October and the appeal panel will have their meeting on the 25th of November.

Further details can be found by clicking the button below. To register to attend the appeal meeting as an observer, click the second button.