Catalyst Pharmaceuticals has announced that Health Canada has accepted their New Drug Submission (NDS) for AGAMREE (vamorolone) with priority review status for treating Duchenne muscular dystrophy (DMD). If approved, AGAMREE would become the first and only Health Canada-approved therapy for DMD, with potential marketing authorization by the end of 2025.
The submission follows AGAMREE’s approvals in the US (2023) and European Union (early 2024). According to the Canadian Neuromuscular Disease registry, over 800 boys and young men in Canada live with DMD, a rare disease causing muscle weakness due to lack of dystrophin protein. (What is Agamree?)
The drug’s development included clinical trials across five Canadian sites, contributing to its global approval. Patient organizations, including Defeat Duchenne Canada and Muscular Dystrophy Canada, have welcomed this development, highlighting the current lack of approved treatments in Canada.
