Dec 18, 2023, AGAMREE (vamorolone) has been approved in the European Union (EU) for the treatment of DMD in patients 4 years of age and older.
Duchenne muscular dystrophy (DMD) is a genetic muscle disease. And The feature is contraction-induced myofibre injury and inflammation. Researchers found that NF-κB signaling is active in dystrophin deficient muscle. Therefore, Inhibiting NF-κB is a potent strategy for the treatment of DMD. Vamorolone is an investigational drug that was developed as a novel dissociative steroidal anti-inflammatory. This means that its chemical profile is such that it shows chemical separation of efficacy (clinical benefit) from some safety concerns (side effect profiles) typically seen with corticosteroids.
AGAMREE is the only approved medication in the EU for treating DMD, and the first DMD treatment approved in both the U.S. and EU.