Vamorolone – a corticosteroid approved by global regulators for the treatment of Duchenne muscular dystrophy. Literature review

Duchenne muscular dystrophy (DMD) is an X-linked hereditary disease that manifests in early childhood and progresses with age, leading to loss of mobility and respiratory and cardiac failure. The globally recognized gold standard for DMD treatment is corticosteroids. However, until recently, only deflazacort (Emflaza) had received approval from the U.S. Food and Drug Administration (FDA) for the indication of DMD. All other drugs in this group were used off-label. At the end of 2023, the FDA, followed by the European Medicines Agency (EMA), registered another drug from the corticosteroid group – vamorolone (Agamree). In 2024, vamorolone received approval in the United Kingdom and China. Thus, all leading global regulators currently recognize vamorolone as a corticosteroids for the treatment of DMD.