Your Monthly Medicines Roundup for March 2026

We have quite a few updates for you this March: the initiation of the assessments for Barth Syndrome and MPS3a; three progress updates and two final recommendations for ARG1d and Friedreich’s Ataxia.  

If you find yourself wanting explanations of the updates or needing a listening ear, you can reach out to us via 08006523181 or email us at contact@metabolicsupport.org.  

On March 4, the consultation period for the pegzilarginase for the treatment of arginase-1 deficiency officially ended. No challenges to the final recommendation were received. With that, the final guidance recommend pegzilarginase for the treatment of people with arginase-1 deficiency by NICE in England. We expect pegzilarginase to be available to the community by early June and have already understood some community members have been given access. If you have any questions or comments, please do get in touch with us. 

Further details about the assessment can by clicking the first button. Our full news article about this development can be accessed via the second button.

In early March, the assessment of Elamipretide for treating Barth syndrome in people of any age was started by NICE in England. Barth Syndrome UK, with support from Metabolic Support, responded to the scoping consultation. We will be providing updates for this assessment as they become available. Further details about the assessment can be found here: 

Further details about the assessment can be found here:

Omaveloxolone for treating Friedreich’s ataxia in adults and adolescents aged 16 years and older was not recommended by SMC in Scotland in March 2026. SMC detailed that the justification provided by the company for the treatment cost was not sufficient compared to the health benefits. They also detailed that the economic analysis was not sufficient for SMC. A Patient and Clinical Engagement meeting did occur, during which patients and clinicians shared their experiences with the disorder and the treatment.

Further details can be found here: 

Since 2023, NICE in England have been tracking venglustat for treating type 3 Gaucher disease in people aged 12 and over after at least 3 years of enzyme replacement therapy. In early March, NICE detailed that the company has notified them that timelines for the assessment will be available soon.

In the meantime, people can track the progress on the NICE website:

The first committee meeting date for this assessment by NICE in England has been published. People interested in attending the meeting on the 23rd of April as an observer can register via the NICE website.  

People can register via the first button. Further details about this appraisal can be found via the second button.

The assessment of rebisufligene etisparvovec for treating mucopolysaccharidosis type IIIA was initiated by NICE in England on the 4th of March. MPS Society is the dedicated patient organisation working on this assessment.

Further details about this assessment can be found here:

Burosumab for treating X-linked hypophosphataemia with radiographic evidence of bone disease in adults is expected to be reassessed by SMC in Scotland this year. In the last assessment, SMC did not recommend burosumab for people with XLH. XLH UK  is the dedicated patient organisation working on this assessment.

Further details can be found here: