10 December 2024: The National Institute for Health and Care Excellence (NICE) has approved vamorolone (Agamree, Santhera) as a treatment option for Duchenne Muscular Dystrophy (DMD) in individuals aged 4 years and over, following a revised pricing agreement with the manufacturer. Despite initial rejection in March due to uncertainties in evidence and cost-effectiveness, further analyses and a revised discount secured its approval. This follows MHRA approval in January.
Approximately 1700 individuals in England with DMD could benefit from this new treatment option. Read the full guidance.
Key Benefits of Vamorolone:
- Alternative to Corticosteroids: Vamorolone serves as an alternative to traditional corticosteroids like prednisone, which are associated with side effects such as osteoporosis and increased fracture risk.
- Potentially Safer Profile: Vamorolone’s unique structure reduces adverse effects on growth and bone health, making it a potentially safer option, especially for patients transitioning from other corticosteroids.
The approval is supported by data from the VISION-DMD clinical trial, a phase 2b, double-blind study involving 121 children with DMD aged 4-6 years.
Key findings included:
- Improved time-to-stand velocity over 24 weeks.
- Minimal impact on height percentile and bone turnover markers compared to prednisone.
Vamorolone could offer important benefits to patients and their families because of its potential to reduce adverse events associated with current steroid treatments
Helen Knight, Director of Medicines Evaluation at NICE
