Vamorolone is recommended, within its marketing authorisation, as an option for treating Duchenne muscular dystrophy (DMD) in people 4 years and over. Vamorolone is only recommended if the company provides it according to the commercial arrangement.
Why the committee made this recommendation
Current treatment options for DMD are limited. Corticosteroids such as prednisone are used to slow progression of the condition.
Evidence from a clinical trial shows that vamorolone improves muscle function compared with placebo. But, it is uncertain whether vamorolone is similar at improving muscle function outcomes, and how well it works in the long term, compared with currently available corticosteroids. Compared with prednisone, the evidence suggests that vamorolone is likely to have fewer side effects, although to what extent is uncertain.
When considering the condition's severity, and its effect on quality and length of life, the most likely cost-effectiveness estimates for vamorolone are within the range that NICE considers an acceptable use of NHS resources. So, it is recommended.