Your Monthly Medicines Roundup for November 2025
Your Monthly Medicines Roundup for November 2025
We have some positive updates to share this month, including the approval of a medicine in Scotland, progression of a medicine and device through the English NICE process and unfortunately a paused appraisal in England.
Familial chylomicronaemia syndrome
Olezarsen, a medicine which can treat familial chylomicronaemia syndrome (FCS), is progressing through the NICE process in England. The invitation to participate in the appraisal has now been shared with all stakeholders and a deadline has been set. Within this scope, we are working with Action FCS and have put together a survey to collect people’s experiences.
If you would like to share your experiences, please click the button below. If you would like to learn more about olezarsen click the second button:
Liver transplant
The scope of the ex-situ machine perfusion devices for deceased donor liver transplants, being undertaken by NICE in England, has been updated to reflect that one of the devices considered in the appraisal will not be made available to the NHS. As multiple developers of the device are being considered, this does not impact the progress of the appraisal.
Further details can be found by clicking the button below:
Nephropathic Cystinosis
As announced earlier this month, we are pleased to share that after 8 years of work, together with Cystinosis Foundation UK, and more recently, Kidney Research UK. Mercaptamine for the treatment of nephropathic cystinosis has now been approved by the Scottish Medicines Consortium in Scotland.
Further details can be found by clicking the buttons below:
Thymidine kinase 2 deficiency
Doxecitine–doxribtimine for treating people with thymidine kinase 2 deficiency at any age has unfortunately been paused by the developing company. This means that the scheduled timelines, including a committee meeting planned for 19 March, will not go ahead. NICE in England will provide an update as soon as new timelines are available. Metabolic Support UK, has been working together with the Lily Foundation and Muscular Dystrophy UK on this appraisal and we will continue to do so once the appraisal is restarted.
Further details can be found by clicking the button below:
