The FDA has granted approval to Catalyst's Agamree (vamorolone), a treatment for Duchenne Muscular Dystrophy (DMD) for patients aged two years and older.
The approval was based on data from the phase 2b VISION-DMD study, supplemented with safety data from three open-label studies. Agamree was administered at various doses over a period of up to 48 months, demonstrating comparable efficacy to standard corticosteroids while showing a reduction in adverse events related to bone health, growth trajectory, and behavior. It is expected to be launched in the first quarter of 2024.
Agamree's unique mode of action stems from its differential effects on glucocorticoid and mineralocorticoid receptors, making it a better-tolerated corticosteroid option. The drug is poised to provide an alternative to the current standard of care for DMD.