Action Duchenne welcomes the exciting news from the Scottish Medicines Consortium’s (SMC) positive recommendation for vamorolone for the treatment of Duchenne muscular dystrophy (DMD) for patients aged 4 years and older in Scotland. This decision follows the National Institute for Health and Care Excellence (NICE) recommendation for vamorolone in England and Wales in late 2024.
This milestone marks a significant step forward in expanding treatment options for children and young adults living with DMD in Scotland, and across the UK.
The clinical trial data suggests that vamorolone may be as effective as corticosteroids in slowing down the loss of motor function, and may have a better side effect profile compared, potentially leading to less growth suppression and improved bone health.
Equitable Access Across the UK
The SMC’s recommendation is a crucial step towards ensuring equitable access to vamorolone across the entire United Kingdom. Action Duchenne applauds the collaborative efforts of the SMC, NICE, and the broader healthcare community in prioritising the needs of those living with DMD.
Through participation in the SMC’s Patient and Clinician Engagement (PACE) and committee meetings, Action Duchenne, alongside Muscular Dystrophy UK and Duchenne UK, emphasised the critical need for improved treatment options. By sharing the lived experiences and challenges of the Duchenne community, our collective advocacy helped shape this positive decision.
Scotland
Vamorolone is now approved and will be made available for prescribing on the NHS in Scotland for patients aged 4 years old or older. Families are encouraged to discuss with your healthcare professional whether this treatment is appropriate for their specific needs.
England and Wales
Following the publication of the Final Draft Guidance by the NICE, the NHS England has up to three months to make vamorolone available to patients.
The Welsh government has also directed the NHS in Wales to implement the guidance within two months of its publication.
Northern Ireland
We have requested clarification from the Department of Health regarding the availability of vamorolone for patients living in Northern Ireland following the NICE and SMC approvals. Updates will be provided as soon additional information becomes available.
About Vamorolone (Agamree)
Vamorolone (often referred to as AGAMREE) is a novel ‘dissociative’ steroid, an anti-inflammatory agent similar to the currently available corticosteroids. It binds to the same receptors as traditional corticosteroids but modifies the downstream activation pathways, aiming to reduce side effects like bone-weakening (osteoporosis). This innovation allows patients to benefit from its anti-inflammatory properties while reducing the risks associated with long-term corticosteriod use. For further insights into vamorolone, explore:
Vamorolone and Corticosteroids – An Overview – Action Duchenne
AGAMREE® (vamorolone) – santhera
Patient Information Leaflet (PIL)
Supporting the Duchenne Community
This development may raise questions within the community. Action Duchenne will continue working tirelessly to ensure access and support for all affected by DMD across the UK. If you have any questions or need support, please reach out to us at info@actionduchenne.org.
