EMFLAZA (deflazacort)
Emflaza is a corticosteroid used for patients with Duchenne muscular dystrophy (DMD) who are two years of age or older. This medication helps patients with the physical challenges of DMD by utilizing its anti-inflammatory properties. It is prescribed to manage this specific type of muscular dystrophy in both pediatric and adult populations.
How EMFLAZA Works
This medication is a prodrug that converts into an active metabolite, which then binds to glucocorticoid receptors. This process works by triggering anti-inflammatory and immunosuppressive responses within the body. Although the exact way these actions improve Duchenne muscular dystrophy is unknown, the drug's primary activity stems from these receptor interactions.
Details
- Status
- Prescription
- First Approved
- 2017-02-09
- Patent Cliff
- 2026
- Routes
- ORAL
- Dosage Forms
- TABLET, SUSPENSION
EMFLAZA Approval History
What EMFLAZA Treats
1 indicationsEMFLAZA is approved for 1 conditions since its original approval in 2017. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Duchenne Muscular Dystrophy
EMFLAZA 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 EMFLAZA's indications
See all emerging drugs →Phase 3 candidates targeting molecules with no FDA-approved drug, in indications EMFLAZA treats. First-in-class if their pivotal trials read out positive.
Drugs Similar to EMFLAZA
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.
EMFLAZA FDA Label Details
Indications & Usage
FDA Label (PDF)EMFLAZA is indicated for the treatment of Duchenne Muscular Dystrophy.
EMFLAZA Patents & Exclusivity
Exclusivity
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Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.