deflazacort

Deflazacort is a synthetic corticosteroid prodrug used primarily to treat Duchenne muscular dystrophy (DMD), a rare inherited muscle disease caused by the absence of dystrophin protein. After oral administration, deflazacort is rapidly metabolized to its active form, 21-desacetyldeflazacort, which binds to glucocorticoid receptor to exert anti-inflammatory and immunosuppressive effects. The drug was first introduced in 1969 for treating various inflammatory conditions and received FDA approval specifically for DMD in February 2017. Deflazacort has a more favorable safety profile compared to other corticosteroids, with less impact on calcium metabolism, growth rate, and bone density. It also has comparatively smaller effects on carbohydrate metabolism, sodium retention, and potassium levels.

Brand names

EmflazaCalcort

Other names

deflazacortdeflazacort tablets