Deflazacort (4 brand names, 2 dosage forms, 1 route)
Available dosage forms: SUSPENSION, TABLET.
Routes of administration: ORAL.
Brand Names
- DEFLAZACORT
- EMFLAZA
- JAYTHARI
- PYQUVI
Indications and Usage
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.
Contraindications
Deflazacort 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 [see Warnings and Precautions (5.15)and Adverse Reactions (6.2)] .
Adverse Reactions
The following serious adverse reactions are discussed in more detail in other sections: Alterations in Endocrine Function [see Warnings and Precautions (5.1)] Immunosuppression and Increased Risk of Infection [see Warnings and Precautions (5.2)] Alterations in Cardiovascular/Renal Function [see Warnings and Precautions (5.3)] Gastrointestinal Perforation [see Warnings and Precautions (5.4)] Behavioral and Mood Disturbances [see Warnings and Precautions (5.5)] Effects on Bones [see Warnings and Precautions (5.6)] Ophthalmic Effects [see Warnings and Precautions (5.7)] Immunizations [see Warnings and Precautions (5.8)] Serious Skin Rashes [see Warnings and Precautions (5.9)] Effects on Growth and Development [see Warnings and Precautions (5.10)] Myopathy [see Warnings and Precautions (5.11)] Kaposi’s Sarcoma [see Warnings and Precautions (5.12)] Thromboembolic Events [see Warnings and Precautions (5.14)] Anaphylaxis [see Warnings and Precautions (5.15)]
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.
Overdosage
Treatment of acute overdosage is by immediate gastric lavage or emesis followed by supportive and symptomatic therapy. For chronic overdosage in the face of severe disease requiring continuous steroid therapy, the dosage of deflazacort may be reduced temporarily, or alternate day treatment may be introduced.