practicalneurology.com
Catalyst Pharmaceuticals reported that Agamree (vamorolone; Coral Gables, FL), an oral suspension approved for the treatment of Duchenne Muscular Dystrophy (DMD) in patients aged > 2 years is now available by prescription in the United States and dispensed through a specialty pharmacy network. Agamree was approved by the Food and Drug Administration in October 2023 as the first-in-class “dissociative steroidal anti-inflammatory” that binds to the same receptors as corticosteroids and modifies their downstream activity. Compared to current standard-of-care corticosteroids, Agamree showed consistent efficacy data and a favorable safety and tolerability profile.
The FDA approval was based on favorable efficacy and safety data from the phase 2b VISION-DMD clinical trial (NCT03439670). Results from trial indicated that participants treated with Agamree showed an improvement of .06 rises per second in the time to stand test (TTSTAND) velocity relative to placebo (95% CI, .02-.10; P=.002). Agamree also met its secondary endpoints, including TTSTAND velocity for the low-dose group, 6-minute walk test for the high- and low-dose group, and time to run/walk in 10 minutes test for the high-dose group.
“This significant milestone offers hope for improved quality of life for individuals living with this devastating rare disease, as current steroid treatment options often involve a significant side effect burden," stated Richard J. Daly, CEO of Catalyst. "With the potential to extend ambulation and mobility for patients, AGAMREE marks an important therapeutic advancement with the prospect of reshaping the treatment paradigm for this life-threatening condition.”
Included in this announcement were details related to “Your Catalyst Pathways” (www.yourcatalystpathways.com)--a comprehensive patient support program for eligible patients.