FDA Approves AGAMREE® (vamorolone) Steroid for the Treatment of Duchenne Muscular Dystrophy

The U.S. FDA announced today its decision to approve AGAMREE (vamorolone), a new steroid for individuals with Duchenne muscular dystrophy 2 years or older.

This approval comes largely based on data from the VISION-DMD study and three open-label studies carried out by ReveraGen Biopharma, which showed that boys on vamorolone had comparable efficacy to current standard-of-care corticosteroids. Data also suggested a reduction in adverse events, notably related to bone health, growth trajectory, and behavior.

“CureDuchenne is proud to have contributed to the early development of vamorolone at ReveraGen, recognizing its potential to overcome some of the challenges related to steroid treatment, such as growth stunting and steroid-induced osteoporosis,” CureDuchenne founder and CEO Debra Miller said. “Reducing these side effects could have significant impact on the quality of life for patients with Duchenne, a paramount goal for CureDuchenne.”

Catalyst Pharmaceuticals, which acquired a license for the treatment in the U.S., Canada, and Mexico, plans to launch AGAMREE in the U.S. by the first quarter of next year. In anticipation of that, Catalyst plans to start accepting enrollment forms in Catalyst Pathways, their patient support program, on December 1, 2023. The enrollment form will be available on YourCatalystPathways.com at that time.

Vamorolone was also recommended for approval in Europe earlier this month, and if successfully approved will be marketed there by Santhera Pharmaceuticals.

To learn more about vamorolone and the recent decision by the FDA, please read Catalyst’s press release here.