Deflazacort

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.

• Deflazacort oral suspension is 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) oral suspension.

• Deflazacort oral suspension 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) ] .contraindicated

The recommended once-daily dosage is approximately 0.9 mg/kg/day administered orally ( 2.2 ) Discontinue gradually when administered for more than a few days ( 2.3 ) 2.1 Assessments Prior to First Dose of Deflazacort Oral Suspension Administer all immunizations according to immunization guidelines prior to starting deflazacort oral suspension. Administer live-attenuated or live vaccines at least 4 to 6 weeks prior to starting deflazacort oral suspension [see Warnings and Precautions (5.8) ]. 2.2 Dosing Information The recommended oral dosage of deflazacort oral suspension is approximately 0.9 mg/kg/day once daily. If the oral suspension is used, round up to the nearest tenth of a milliliter (mL). 2.3 Discontinuation Dosage of deflazacort oral suspension must be decreased gradually if the drug has been administered for more than a few days [see Warnings and Precautions (5.1 )]. 2.4 Important Preparation and Administration Instructions Deflazacort oral suspension can be taken with or without food. Do not administer deflazacort oral suspension with grapefruit juice [see Drug Interactions (7.1) ]. Shake deflazacort oral suspension well before administration. Use only the oral dispenser provided with the product. After withdrawing the appropriate dose into the oral dispenser, slowly add the deflazacort oral suspension into 3 to 4 ounces of juice (except grapefruit juice) or milk and mix well. The dose should then be administered immediately. Discard any unused deflazacort oral suspension remaining after 1 month of first opening the bottle.

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 ( 5.2 ) Alterations in Cardiovascular/Renal Function : Monitor for elevated blood pressure and sodium, and for decreased potassium levels ( 5.3 ) Gastrointestinal Perforation: Increased risk in patients with certain GI disorders; Signs and symptoms may be masked ( 5.4 ) Behavioral and Mood Disturbances: May include euphoria, insomnia, mood swings, personality changes, severe depression, and psychosis ( 5.5 ) Effects on Bones: Monitor for decreases in bone mineral density with chronic use of deflazacort ( 5.6 ) Ophthalmic Effects: May include cataracts, infections, and glaucoma; Monitor intraocular pressure if deflazacort is continued for more than 6 weeks ( 5.7 ) Vaccination: Do not administer live or live attenuated vaccines to patients receiving immunosuppressive doses of corticosteroids.

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) ] Risk of Serious Adverse Reactions in Infants because of Benzyl Alcohol Preservative [see Warnings and Precautions (5.13) ] Thromboembolic Events [see Warnings and Precautions (5.14) ] Anaphylaxis [see Warnings and Precautions (5.15) ] The most common adverse reactions (≥10% for deflazacort and greater than placebo) are Cushingoid appearance, weight increased, increased appetite, upper respiratory tract infection, cough, pollakiuria, hirsutism, central obesity, and nasopharyngitis ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Zydus Pharmaceuticals (USA) Inc. at 1-877-993-8779 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch .

8.1 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. There are no adequate and well-controlled studies with deflazacort in pregnant women to inform drug-associated risks. Corticosteroids, including deflazacort, readily cross the placenta. Adverse developmental outcomes, including orofacial clefts (cleft lip, with or without cleft palate) and intrauterine growth restriction, and decreased birth weight, have been reported with maternal use of corticosteroids, including deflazacort, during pregnancy. Some epidemiologic studies report an increased risk of orofacial clefts from about 1 per 1,000 infants to 3 to 5 per 1,000 infants; however, a risk for orofacial clefts has not been observed in all studies. Intrauterine growth restriction and decreased birth weight appear to be dose-related; however, the underlying maternal condition may also contribute to these risks (see Data) . The estimated background risk of major birth defects and miscarriage for the indicated populations is unknown. In the U.S.

Metabolism

Absorption After oral administration in the fasted state, the median T max with deflazacort suspension is about 1 hour (range 0.25 to 2 hours). Food Effect: Co-administration of deflazacort tablets with a high-fat meal reduced C max by about 30% and delayed T max by one hour, relative to administration under fasting conditions, but there was no effect on the overall systemic absorption as measured by AUC.

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.

• Feb 9, 2017NDANDA208684Ptc Therap
• Feb 9, 2017NDANDA208685Ptc Therap
• Feb 9, 2024ANDAANDA217123Aurobindo Pharma Ltd
• Apr 25, 2024ANDAANDA217813Tris Pharma Inc
• Nov 5, 2024ANDAANDA216720Upsher Smith Labs
• Mar 18, 2025ANDAANDA217741Sun Pharm
• Apr 8, 2025ANDAANDA219254Zydus Lifesciences
• Jun 30, 2025ANDAANDA219417Aucta

1. 1Weight Increased5288.4%
2. 2Off Label Use4056.5%
3. 3Fall2994.8%
4. 4Product Dose Omission Issue2484.0%
5. 5Vomiting2443.9%
6. 6No Adverse Event2433.9%
7. 7Pain2273.6%
8. 8Pyrexia2203.5%
9. 9Headache2133.4%
10. 10Influenza2093.3%
11. 11Drug Ineffective2083.3%
12. 12Malaise2083.3%
13. 13Weight Decreased1873.0%
14. 14Arthralgia1832.9%
15. 15Diarrhoea1812.9%

The 10 most recently updated of 23 ClinicalTrials.gov registrations naming Deflazacort as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• Mycophenolate Mofetil in Patients With Progressive Idiopathic Membranous Nephropathy
  Completed · Phase 3 · Interventional · 43 enrolled · Kyungpook National University Hospital
  NCT01282073updated 2025-08-27
• Deflazacort Vs. Prednisolone in Acute-stage ABPA
  Active not recruiting · Phase 2 · Phase 3 · Interventional · 150 enrolled · Post Graduate Institute of Medical Education and Research, Chandigarh
  NCT04227483updated 2025-02-28
• Phase 3 Trial of Pamrevlumab or Placebo in Combination With Systemic Corticosteroids in Participants With Ambulatory DMD
  Terminated · Phase 3 · Interventional · 73 enrolled · Kyntra Bio
  NCT04632940updated 2024-08-26
• Phase 3 Trial of Pamrevlumab or Placebo With Systemic Corticosteroids in Participants With Non-ambulatory Duchenne Muscular Dystrophy (DMD)
  Terminated · Phase 3 · Interventional · 98 enrolled · Kyntra Bio
  NCT04371666updated 2024-03-12
• Registry of IgA Nephropathy in Chinese Children
  Unknown · Observational · 1,200 enrolled · Peking University First Hospital
  NCT03015974updated 2023-12-06
• Evaluation of the Effects of Pre-operative Deflazacort on Post-operative Pain, Edema and Trismus
  Unknown · Phase 4 · Interventional · 35 enrolled · Yuzuncu Yil University
  NCT04365088updated 2023-08-22
• The Nationwide Real-world Status of Oral Corticosteroid Use in Patients With Asthma
  Unknown · Observational · 800,000 enrolled · Joon Young Choi
  NCT05750797updated 2023-03-06
• Finding the Optimum Regimen for Duchenne Muscular Dystrophy
  Completed · Phase 3 · Interventional · 196 enrolled · University of Rochester
  NCT01603407updated 2022-08-12
• A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)
  Terminated · Phase 3 · Interventional · 11 enrolled · PTC Therapeutics
  NCT03783923updated 2022-06-27
• Study of the Excretion of Orally Administered Corticosteroids for the Improval of the Detection of Said Substances in Anti-doping Controls
  Unknown · Phase 1 · Interventional · 50 enrolled · Parc de Salut Mar
  NCT04791345updated 2021-04-01

Frequently asked questions

How does Deflazacort work?

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.

What is Deflazacort used for?

According to FDA labeling, Deflazacort carries indications including: Deflazacort oral suspension is 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) oral suspension.. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Deflazacort?

Deflazacort is classified as Glucocorticoids, Corticosteroid, Corticosteroid Hormone Receptor Agonists, Glucocorticoid Receptor Agonists, Decreased Immunologic Activity.

What are the brand names for Deflazacort?

Deflazacort is marketed under brand names including Emflaza, Jaythari, Kymbee, Pyquvi.

What are the contraindications for Deflazacort?

Deflazacort labeling lists contraindications including: Deflazacort oral suspension 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) ] .. Always consult the full prescribing information and a clinician.