vamorolone

Vamorolone is a novel, fully synthetic corticosteroid developed for the treatment of Duchenne muscular dystrophy (DMD), a rare inherited muscle disease caused by the absence of dystrophin. It is classified as a dissociative steroid and acts primarily as an agonist at the glucocorticoid receptor to exert anti-inflammatory and immunosuppressive effects. Unlike traditional corticosteroids, vamorolone also antagonizes the mineralocorticoid receptor and has been designed to reduce some of the adverse side effects associated with classic steroids such as prednisolone or deflazacort. Its unique structure lacks an 11-β oxygen moiety, making it less susceptible to certain metabolic pathways that contribute to side effects like osteoporosis. Vamorolone was approved in October 2023 under the brand name Agamree for use in patients aged 2 years and older with DMD[1][3][5][6][7].

Brand names

Agamree

Other names

vamoroloneVBP15VBP-15VBP 15Agamree