The Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion in favor of the approval of AGAMREE® (vamorolone) for the treatment of Duchenne Muscular Dystrophy (DMD) patients aged 4 years and older.
Vamorolone is a novel drug candidate that offers an alternative to existing corticosteroids (e.g. Prednisone, Deflazacort), the current standard of care in children and adolescent patients with DMD. AGAMREE® (vamorolone) was co-created with the full collaboration of DMD patient organizations in every stage of the development process as funders, active research participants, and the collaboration of World Duchenne Organization (WDO) as a partner in the VISION-DMD Project.
The development of AGAMREE® (vamorolone) is the response to the high unmet medical need for an effective and well-tolerated treatment for all DMD patients, particularly paediatric patients.
The CHMP’s positive opinion recognizes the clinical significance of AGAMREE in addressing this need, including the noteworthy safety benefits compared to the standard corticosteroid care for DMD and paves the way for a potential European Commission (EC) decision on marketing authorization in late 2023. This decision holds the promise of AGAMREE becoming the first drug fully approved by the European Medicines Agency (EMA) for the treatment of patients with DMD. Furthermore, it also highlights the importance of a new patient centered model for drug development in rare diseases.
In parallel, the United States is also anticipating approval of vamorolone with the Prescription Drug User Fee Act (PDUFA). The action date is set for October 26, 2023, opening the door for AGAMREE’s potential launch in the U.S. in Q1-2024.