DEFLAZACORT
Deflazacort is a corticosteroid used for patients with Duchenne muscular dystrophy (DMD) who are five years of age or older. It helps patients with this condition by utilizing its anti-inflammatory and immunosuppressive properties. This medication is prescribed to manage the symptoms of DMD in both pediatric and adult populations within the approved age range.
How DEFLAZACORT Works
Deflazacort works by converting into an active metabolite known as 21-desDFZ, which targets and binds to glucocorticoid receptors. This interaction triggers anti-inflammatory and immunosuppressive effects within the body. Although the specific process by which these actions improve Duchenne muscular dystrophy is not yet fully known, the drug relies on these receptor-mediated pathways to function.
Details
- Status
- Prescription
- First Approved
- 2024-02-09
- Routes
- ORAL
- Dosage Forms
- SUSPENSION, TABLET
DEFLAZACORT Approval History
What DEFLAZACORT Treats
1 indicationsDEFLAZACORT is approved for 1 conditions since its original approval in 2024. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Duchenne Muscular Dystrophy
DEFLAZACORT Competitive Set
ProThree rings of competition based on shared molecular targets and treated indications.
Direct competitors
Same target(s) AND same indication — head-to-head.
MoA expansion candidates
Same target(s), different indications — where else is this mechanism being explored?
Indication competitors
Same indication, different mechanism — what else might this patient receive?
Filters applied: drops same-active-ingredient (505(b)(2) reformulations), route-mismatch (topical vs systemic), and cross-therapeutic-area matches in same-indication rings.
What's emerging in DEFLAZACORT's indications
See all emerging drugs →Phase 3 candidates targeting molecules with no FDA-approved drug, in indications DEFLAZACORT treats. First-in-class if their pivotal trials read out positive.
Drugs Similar to DEFLAZACORT
3 of 9FDA-approved drugs for similar conditions. Compare mechanisms and indications to understand treatment alternatives.
Clinical Trial Registry
9 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT04227483 | RA/2020/001 | Ph 2, Ph 3 | active not recruiting | Deflazacort Vs. Prednisolone in Acute-stage ABPA |
| NCT01603407 FOR-DMD results posted | U01NS061799 2010-023744-33, U01NS061799 | Ph 3 | completed | Finding the Optimum Regimen for Duchenne Muscular Dystrophy |
| NCT03783923 results posted | PTCEMF-GD-004 | Ph 3 | terminated | A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I) |
| NCT03642145 PTCEMF | PTCEMF-GD-003 | Ph 3 | withdrawn | A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD) |
| NCT02295748 | MP-104-CL-022OLE | Ph 1 | completed | An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability Deflazacort |
| NCT02485431 | MP-104-CL-026 | Ph 1 | completed | Food Effect and Bioavailability of Deflazacort Formulations in Healthy Volunteers |
| NCT02251600 | MP-104-CL-005 | Ph 1 | completed | A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy |
| NCT02286622 | MP-104-CL-024 | Ph 1 | completed | A Single Dose Evaluation of the Effects of Renal Impairment on Deflazacort Pharmacokinetics |
| NCT02286609 | MP-104-CL-023 | Ph 1 | completed | A Single Dose Evaluation of the Effects of Moderate Hepatic Impairment on Deflazacort Pharmacokinetics |
Active Pipeline
Ongoing clinical trials by development phase
Key Completed Trials
Completed studies with published results, ranked by significance
Trial Timeline
Full development history with FDA approval milestones
Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
DEFLAZACORT FDA Label Details
Indications & Usage
DEFLAZACORT is indicated for the treatment of Duchenne Muscular Dystrophy.
Looking for the branded version?
EMFLAZA
Full clinical data, patents, trials, and competitive landscape for deflazacort.
Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.