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Emflaza (DEFLAZACORT)
Emflaza works by mimicking the effects of cortisol, a natural hormone produced by the body, to reduce inflammation and suppress the immune system.
Emflaza (DEFLAZACORT) is a corticosteroid medication originally developed by Marathon Pharmaceuticals LLC and currently owned by PTC Therapeutics. It is a small molecule that has been FDA-approved since 2017 for the treatment of Duchenne muscular dystrophy. Emflaza is available as a generic medication, with multiple manufacturers, and is no longer under patent protection. As a corticosteroid, it works by suppressing the immune system and reducing inflammation. Key safety considerations include potential side effects such as weight gain, increased blood pressure, and changes in glucose and lipid metabolism.
At a glance
| Generic name | DEFLAZACORT |
|---|---|
| Sponsor | Ptc Therap |
| Drug class | Corticosteroid [EPC] |
| Modality | Small molecule |
| Therapeutic area | Immunology |
| Phase | FDA-approved |
| First approval | 2017 |
Mechanism of action
Deflazacort is 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.
Approved indications
- Duchenne muscular dystrophy
Common side effects
- Cushingoid appearance
- Weight increased
- Increased appetite
- Upper respiratory tract infection
- Cough
- Pollakiuria
- Nasopharyngitis
- Hirsutism
- Central obesity
- Erythema
- Irritability
- Rhinorrhea
Key clinical trials
- Mycophenolate Mofetil in Patients With Progressive Idiopathic Membranous Nephropathy (PHASE3)
- Deflazacort Vs. Prednisolone in Acute-stage ABPA (PHASE2,PHASE3)
- Phase 3 Trial of Pamrevlumab or Placebo in Combination With Systemic Corticosteroids in Participants With Ambulatory DMD (PHASE3)
- Phase 3 Trial of Pamrevlumab or Placebo With Systemic Corticosteroids in Participants With Non-ambulatory Duchenne Muscular Dystrophy (DMD) (PHASE3)
- Registry of IgA Nephropathy in Chinese Children
- Evaluation of the Effects of Pre-operative Deflazacort on Post-operative Pain, Edema and Trismus (PHASE4)
- The Nationwide Real-world Status of Oral Corticosteroid Use in Patients With Asthma
- Finding the Optimum Regimen for Duchenne Muscular Dystrophy (PHASE3)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| FDA label | Mechanism, indications, dosing, boxed warnings, drug interactions |
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Emflaza CI brief — competitive landscape report
- Emflaza updates RSS · CI watch RSS
- Ptc Therap portfolio CI