Drug updated on 4/16/2024

Dosage FormSuspension (oral; 40 mg/mL)
Drug ClassCorticosteroids
Ongoing and
Completed Studies


  • Indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 2 years of age and older.

Latest News

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  • 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‚Äôs 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 TitleYearSource
Agamree (vamorolone) Prescribing Information. 2023Santhera Pharmaceuticals (USA), Inc., Burlington MA

Systematic Reviews / Meta-Analyses