following a full submission assessed under the orphan medicine process:
vamorolone (Agamree®) is accepted for use within NHSScotland.
Indication under review: treatment of Duchenne muscular dystrophy (DMD) in patients aged 4 years and older.
In a randomised, double-blind, phase IIb study, treatment with vamorolone resulted in a significant improvement in the change in time to stand from supine (TTSTAND) velocity and change in 6-minute walk test (6MWT) distance between baseline and week 24, compared with placebo.
This advice applies only in the context of an approved NHSScotland Patient Access Scheme (PAS) arrangement delivering the cost-effectiveness results upon which the decision was based, or a PAS/ list price that is equivalent or lower.
This advice takes account of the views from a Patient and Clinician Engagement (PACE) meeting.
Medicine details
- Medicine name:
- vamorolone (Agamree)
- SMC ID:
- SMC2721
- Indication:
For the treatment of Duchenne muscular dystrophy (DMD) in patients aged 4 years and older.
- Pharmaceutical company
- Santhera Pharmaceuticals
- BNF chapter
- Musculoskeletal and joint diseases
- Submission type
- Full
- Status
- Accepted
- Date advice published
- 13 January 2025