PYQUVI (deflazacort)
PYQUVI is a corticosteroid used for patients 5 years of age and older who have Duchenne muscular dystrophy (DMD). It helps patients with this condition by providing systemic anti-inflammatory and immunosuppressive support. This medication is prescribed to manage the effects of DMD in both pediatric and adult populations.
How PYQUVI Works
This drug works by converting into an active metabolite that targets glucocorticoid receptors to produce anti-inflammatory and immunosuppressive effects. While these biological actions are clear, the specific process by which they provide therapeutic benefits to patients with Duchenne muscular dystrophy is not yet fully understood.
Development Insights
Details
- Status
- Prescription
- First Approved
- 2025-06-30
- Routes
- ORAL
- Dosage Forms
- SUSPENSION
PYQUVI Approval History
What PYQUVI Treats
1 indicationsPYQUVI is approved for 1 conditions since its original approval in 2025. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Duchenne Muscular Dystrophy
PYQUVI 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 PYQUVI's indications
See all emerging drugs →Phase 3 candidates targeting molecules with no FDA-approved drug, in indications PYQUVI treats. First-in-class if their pivotal trials read out positive.
Drugs Similar to PYQUVI
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.
PYQUVI FDA Label Details
Indications & Usage
PYQUVI is indicated for the treatment of Duchenne Muscular Dystrophy.
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Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.
How We Calculate These Metrics
Trial Activity Stage
Measures the current development activity pattern based on trial phases, status, and trends. Important: This measures R&D activity, not commercial lifecycle.
Trial statuses: "Active" means recruiting or ongoing. "Completed" means reached planned endpoint. "Terminated" means stopped early—often due to safety, efficacy, or business reasons.
- Growth: High proportion of early-phase trials (Phase 1/2), active development
- Expansion: Significant Phase 3 activity, approaching or pursuing approvals
- Mature: Substantial Phase 4 post-marketing studies
- Stable: Mixed phase distribution, steady development
- Declining: Low active trial ratio, reduced R&D investment