FDA Approves Agamree (vamorolone) for the Treatment of Duchenne Muscular Dystrophy
Catalyst Pharmaceuticals, Inc. has reported that Santhera Pharmaceuticals has obtained U.S. Food and Drug Administration (“FDA”) approval for Agamree® (vamorolone) oral suspension 40 mg/mL for use in treating Duchenne Muscular Dystrophy (DMD) in patients aged two years and older.
Agamree offers a novel corticosteroid treatment option for DMD, addressing a significant unmet medical need.
Agamree was granted Orphan Drug and Rare Pediatric Disease designations status for DMD in the U.S. and will be eligible for seven years of orphan drug exclusivity upon approval date and has issued pending patents that could provide protection until 2040.
In July 2023, Catalyst secured the exclusive North American license and commercial rights for Agamree from Santhera for DMD and other potential indications, bolstering its neuroscience commercial portfolio with a highly synergistic neuromuscular asset. As part of that transaction, Santhera will promptly transfer the approved New Drug Application for Agamree to Catalyst. The company plans to launch the product in Q1 2024.
Agamree’s unique mode of action is based on differential effects on glucocorticoid and mineralocorticoid receptors and modifying further downstream activity and, as such, is considered a novel corticosteroid with dissociative properties in maintaining efficacy, with a better-tolerated side effect profile. This mechanism of action may allow vamorolone to emerge as an effective alternative to the current standard of care corticosteroids in children, adolescents, and adult patients with DMD.
