Understanding your views of vamorolone, a treatment for Duchenne muscular dystrophy currently being appraised by NICE

Vamorolone received approval for use in the UK by the Medicines and Healthcare products Regulatory Agency (MHRA) in January 2024, allowing NICE to now explore whether or not it should recommend that it be made available on the NHS in England.

We are supporting this process as one of two ‘Patient Experts’, and our Director of Care, Campaigns and Support, Rob Burley, will be taking part in the appraisal committee meeting on 5 March 2024 alongside a parent of a child who has experience of receiving vamorolone.  


We have developed a short survey to gather further views and insight from the Duchenne community to ensure that these are fully represented in the appraisal process. 


Vamorolone is manufactured by the company Santhera. In response to approval by the MHRA, the company noted the favorable safety and tolerability profile of vamorolone compared to conventional corticosteroids. Corticosteroids are routinely prescribed for Duchenne muscular dystrophy, as they slow the decline in muscle strength and mobility over time. However, there are many possible side-effects which must be carefully managed.  


The company highlighted results from its VISION-DMD study, which showed that boys treated with vamorolone on average maintained growth similar to those treated with placebo, whilst those treated with prednisone (a corticosteroid) on average experienced growth stunting.  


Patients who switched from prednisone to vamorolone after 24-weeks were, on average, able to resume growing in height over the remainder of the study. Unlike corticosteroids, vamorolone did not result in a reduction of bone metabolism. In addition, patients who switched from a standard of care corticosteroid to vamorolone maintained the efficacy benefit while recovering their growth and bone health. 


The VISION-DMD study did report some adverse side-effects, which included vomiting and vitamin D deficiency. These adverse side-effects were reported as being generally of mild to moderate severity. 


NICE is particularly interested in understanding more about the community’s thoughts on existing corticosteroid treatment options, and the potential advantages or disadvantages that vamorolone may have in comparison to them. 


The survey will close at midnight on Sunday 4 February 2024. We will provide further information and updates as the process continues and if you have any questions or comments you can contact info@musculardystrophyuk.org.