Vamorolone (sold under the brand name Agamree) is a treatment for inflammation associated with Duchenne muscular dystrophy. It is an alternative to other corticosteroids and people may experience fewer side-effects. It is manufactured by the pharmaceutical company Santhera.
Vamorolone
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How it works
Vamorolone binds to the same receptors as corticosteroids but modifies the downstream activity of the receptors. High dose corticosteroids have significant side effects that may detract from patient quality of life. People taking vamorolone may still experience side effects from taking the drug.
Availability
England, Wales and Northern Ireland
Since December 2024, vamorolone has been recommended by the National Institute for Health and Care Excellence (NICE) as an option for treating Duchenne muscular dystrophy (DMD) in people four years and over in England. Integrated Care Boards and NHS England must comply with a NICE recommendation within three months of its date of publication. This means that, if a patient has Duchenne muscular dystrophy and the healthcare professional responsible for their care thinks that vamorolone is the right treatment, it should be available for use in April 2025.
When NICE recommends the use of a treatment, the NHS in Wales must usually provide funding and resources for it within two months of the publication. Northern Ireland also generally follows NICE guidance.
We played a leading role in NICE’s appraisal process, working in partnership with other Duchenne charities. We nominated and supported Mandy Roe as a patient expert to share her 12-year-old son’s experience of receiving the treatment through a clinical trial. Our Director of Care, Campaigns and Support also served as a patient expert to the appraisal process and represented the views of the Duchenne community, which we gathered through extensive engagement.
More information on NICE’s appraisal of vamorolone can be found here.
Scotland
Since January 2025, vamorolone has been accepted by the Scottish Medicines Consortium (SMC) as a treatment option for people aged four years and over in Scotland with Duchenne muscular dystrophy.
We played a leading role in the Scottish Medicines Consortium appraisal process, working in partnership with other Duchenne charities. We supported Gerry McMenemy as a patient expert to share his son’s experience of living with DMD.
More information on SMC’s appraisal of vamorolone can be found here.
Support and information
Duchenne muscular dystrophy
Duchenne muscular dystrophy is a muscle wasting condition caused by the lack of a protein called dystrophin. It usually affects only boys.
Research
We fund groundbreaking research to learn more about muscle wasting conditions and lead us to new treatments. We’ve already made advances that would have been unthinkable just 10 years ago, and we are determined to go even further and faster.
Our support services
We are here for everyone, but we understand that support isn’t one-size-fits-all. Speak to us. We can tailor our support to meet your unique needs.
Our helpline is open Monday-Thursday 10am-2pm.