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Vamorolone for treating Duchenne muscular dystrophy in people 4 years and over

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1 Recommendations

1.1

Vamorolone is not recommended, within its marketing authorisation, for treating Duchenne muscular dystrophy (DMD) in people 4 years and over.

1.2

This recommendation is not intended to affect treatment with vamorolone that was started in the NHS before this guidance was published. People having treatment outside this recommendation may continue without change to the funding arrangements in place for them before this guidance was published, until they and their NHS clinician consider it appropriate to stop. For children or young people, this decision should be made jointly by the clinician, the child or young person, and their parents or carers.

Why the committee made these recommendations

Current treatment options for DMD are limited, and there is an unmet need for new treatments. Corticosteroids, specifically prednisone, prednisolone or deflazacort, are used to slow progression of the condition.

Evidence from a clinical trial shows that vamorolone improves muscle function outcomes compared with placebo. But the trial only included a small number of people and was short. So, compared with prednisone, it is uncertain whether vamorolone is similar at improving muscle function outcomes, and how well it works in the long term. The evidence suggests that vamorolone is likely to reduce the number of side effects compared with prednisone, but to what extent is uncertain. Also, the trial only included people who had not started treatment for DMD. So, it is uncertain how well vamorolone works for people who have had corticosteroids. There are no comparisons with other corticosteroids.

Because of the uncertainties in the clinical evidence and economic model, it is not possible to determine the most likely cost-effectiveness estimates for vamorolone. So, further modelling is needed, and vamorolone is not recommended.