Deflazacort

What it is and how it works

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.

Mechanism class: Corticosteroid Hormone Receptor Agonists

Indications

Deflazacort tablets are indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 5 years of age and older. Additional pediatric use information is approved for PTC Therapeutics, Inc.'s Emflaza™ (deflazacort) tablets. However, due to PTC Therapeutics, Inc.'s marketing exclusivity rights, this drug product is not labeled with that information. Deflazacort is a corticosteroid indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 5 years of age and older.

Dosage and administration (summary)

The recommended once-daily dosage is approximately 0.9 mg/kg/day administered orally. Discontinue gradually when administered for more than a few days. Assessments Prior to First Dose of Deflazacort Tablets Administer all immunizations according to immunization guidelines prior to starting deflazacort tablets. Administer live-attenuated or live vaccines at least 4 to 6 weeks prior to starting deflazacort tablets . Dosing Information The recommended oral dosage of deflazacort tablets is approximately 0.9 mg/kg/day once daily. If tablets are used, round up to the nearest possible dose. Any combination of the four deflazacort tablet strengths can be used to achieve this dose.

Contraindications

Deflazacort tablet is contraindicated in patients with known hypersensitivity to deflazacort or to any of the inactive ingredients. Instances of hypersensitivity, including anaphylaxis, have occurred in patients receiving corticosteroid therapy . Hypersensitivity to deflazacort or any of the inactive ingredients in deflazacort tablets.

Warnings and precautions

Alterations in Endocrine Function: Hypothalamic-pituitary-adrenal axis suppression, Cushing’s syndrome and hyperglycemia can occur; Monitor patients for these conditions with chronic use of deflazacort. ( 2.3 , 5.1 ) Immunosuppression and Increased Risk of Infection : Increased risk of new, exacerbation, dissemination, or reactivation of latent infections, which can be severe and at times fatal; Signs and symptoms of infection may be masked. Alterations in Cardiovascular/Renal Function: Monitor for elevated blood pressure and sodium and for decreased potassium levels.

Pregnancy

Pregnancy Risk Summary Corticosteroids should be used during pregnancy only if the potential benefit justifies the potential risk to the fetus. Infants born to mothers who have received substantial doses of corticosteroids during pregnancy should be carefully observed for signs of hypoadrenalism.

Storage and supply

How Supplied Deflazacort Tablets, 6 mg are supplied as white to off-white, round, uncoated tablet debossed with ‘J6’ on one side and plain on other side. They are available as below: Bottle of 100 Tablets: NDC 60219-2282-1 (1 Bottle in 1 Carton) Deflazacort Tablets, 18 mg are supplied as white to off-white, round, uncoated tablet debossed with ‘AC9…

Source: Deflazacort (Deflazacort) — US FDA prescribing information, 2025-06-16, DailyMed. Summarised and shortened; the full label governs. Iranian registration, indications and labelling may differ.