Santhera Pharmaceuticals has recently announced the approval of AGAMREE® (vamorolone) in the United Kingdom (UK) for the treatment of Duchenne muscular dystrophy (DMD) in patients aged 4 and above, irrespective of their underlying mutation and ambulatory status. The UK's Medicines and Healthcare products Regulatory Agency (MHRA), in line with the European Medicines Agency (EMA), has acknowledged the significant safety benefits of AGAMREE. These benefits include maintaining normal bone metabolism, density, and growth compared to standard corticosteroids.
The approval highlights both the efficacy and safety of AGAMREE in treating DMD, with the MHRA recognizing its favorable safety and tolerability profile compared to conventional corticosteroids. This follows the earlier approvals by the U.S. Food and Drug Administration (FDA) for patients aged 2 and above in the U.S. and by the European Commission for patients aged 4 and above in the EU.
AGAMREE's approval in the UK marks a milestone, making it the first and only fully approved medicinal product for DMD in both the EU and UK. This positions AGAMREE as the initial treatment to receive approval for DMD across all three geographies—U.S., EU, and UK.
Vamorolone, the active component of AGAMREE, operates by binding to the same receptor as glucocorticoids but modifies downstream activity. Importantly, it is not a substrate for the 11-β-hydroxysteroid dehydrogenase (11β-HSD) enzymes, which may contribute to local tissue amplification and corticosteroid-associated toxicity. This unique mechanism of action has the potential to separate efficacy from steroid safety concerns, positioning vamorolone as an alternative to existing corticosteroids, the current standard of care for children and adolescents with DMD.
AGAMREE has received Orphan Drug status for DMD in the U.S. and Europe, as well as Fast Track and Rare Pediatric Disease designations from the U.S. FDA. Additionally, it holds Promising Innovative Medicine (PIM) status from the UK MHRA for DMD. Notably, AGAMREE is now approved for use in the United States, the European Union, and the United Kingdom.
