Deflazacort, also known as Emflaza, is a corticosteroid prodrug used to treat Duchenne muscular dystrophy (DMD). This disease is an inherited disorder resulting from mutations of the dystrophin gene, which is important for muscle function. This causes muscle weakness and progressive breathing and cardiovascular disability. Deflazacort improves various symptoms, including muscle weakness and cardiorespiratory symptoms in addition to delaying their onset. Since deflazacort is a prodrug, it is metabolized to 21-deflazacort by plasma esterases. 21-deflazacort binds to the glucocorticoid receptor to exert anti-inflammatory and immunosuppressive effects on the body. The exact mechanism by which deflazacort exerts its therapeutic effects in patients is still unclear but likely occurs via its anti-inflammatory activities. By binding to the glucocorticoid receptor, it probably influences transcription factors AP-1 and NF-kB to block the transcription of COX-2/prostaglandin G/H synthase 2 which reduces the amount of prostanoids being produced from arachidonic acid. Prostanoids such as PGI2 and thromboxane A2 influence the effects of inflammation through vasoconstriction/dilation, pain sensitivity, and platelet aggregation. 21-deflazacort could also affect the promoter of annexin-1, an important inflammatory protein as it affects leukocytes and blocks phospholipase A2 which reduces the amount of arachidonic acid being cleaved from the phospholipid bilayer. Reducing the amount of arachidonic acid formed further decreases the concentrations of prostanoids mentioned calming inflammation. Deflazacort is administered as an oral suspension or tablet.

Deflazacort Pathway References