AGAMREE (vamorolone) is a novel corticosteroid approved for Duchenne muscular dystrophy (DMD), designed to modulate inflammation while potentially mitigating traditional steroid-related adverse effects. It binds to the glucocorticoid receptor, exerting anti-inflammatory and immunosuppressive actions, though its precise mechanism in DMD remains under study. Unlike conventional corticosteroids, vamorolone demonstrates dissociative properties that may preserve bone health and growth metrics, as suggested by preclinical models. Clinical trials highlight its efficacy in improving functional endpoints such as Time to Stand Test (TTSTAND) velocity and 6-Minute Walk Test (6MWT) distance, with a safety profile characterized by lower incidence of cushingoid features compared to prednisone.

AGAMREE is administered as an oral suspension (40 mg/mL), taken once daily with food to enhance absorption. Unique to its class, it allows direct switching from other corticosteroids (e.g., prednisone) without dose tapering, reducing adrenal crisis risk. Dosing flexibility accommodates hepatic impairment and drug interactions (e.g., strong CYP3A4 inhibitors). Key warnings include immunosuppression, endocrine dysfunction, and behavioral disturbances. Long-term effects on growth, bone density, and cardiac outcomes remain under surveillance, with no geriatric data available due to DMD’s pediatric predominance. Clinical response is monitored via functional assessments and metabolic parameters, aligning with DMD management guidelines.