Daily Medication Pearl: Emflaza (Deflazacort)

Medication Pearl of the Day: Emflaza (Deflazacort)

Indication: Emflaza is a corticosteroid indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients aged 2 years and older.

Insights:

• Dosing: The recommended once-daily dosage is approximately 0.9 mg/kg/day administered orally.
• Dosage forms: Tablets: 6, 18 , 30 , and 36 mg-Oral Suspension: 22.75 mg/mL.
• Adverse events: The most common adverse reactions (≥ 10% for Emflaza and greater than the placebo) are central obesity, cough, Cushingoid appearance, hirsutism, increased appetite, nasopharyngitis, pollakiuria, upper-respiratory-tract infection, and weight increase.
• Mechanism of action: Deflazacort is a corticosteroid prodrug, whose active metabolite, 21-desDFZ, acts through the glucocorticoid receptor to exert anti-inflammatory and immunosuppressive effects. The precise mechanism by which deflazacort exerts its therapeutic effects in patients with DMD is unknown.

Source:

Prescribing Information | EMFLAZA® (deflazacort)