Drug updated on 4/16/2024
| Dosage Form | Suspension (oral; 40 mg/mL) |
| Drug Class | Corticosteroids |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 2 years of age and older.
Summary
- Vamorolone (Agamree) is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 2 years of age and older, showing significant improvements in motor function compared to placebo.
- A systematic review/meta-analysis included data from four studies with a total of 277 DMD patients divided into three groups: vamorolone, glucocorticoids, and placebo.
- Compared to the placebo group, Agamree showed statistically significant improvements in Time to Stand velocity, Time to Run/Walk velocity, and Time to Climb velocity.
- When compared with glucocorticoids therapy for DMD treatment, vamorolone demonstrated an increased height percentile for age, suggesting a potential advantage in growth development along with improvement in motor abilities.
- The safety profile of Agamree remains tentative as explicit data on side effects or long-term outcomes were not provided; however, its comparative efficacy suggests a favorable profile pending further investigation.
- While the drug's target demographic is clear - DMD patients aged 2 years and above - there’s an implied need for further exploration within various subgroups such as different stages of DMD or diverse racial backgrounds within the population.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Agamree (vamorolone) Prescribing Information. | 2023 | Santhera Pharmaceuticals (USA), Inc., Burlington MA |
Systematic Reviews / Meta-Analyses
| Document Title | Year | Source |
|---|---|---|
| Efficacy of vamorolone in treatment of Duchene muscle dystrophy. A meta-analysis. | 2023 | Frontiers in Neurology |