Product Description
Deflazacort is used to treat Duchenne muscular dystrophy (DMD; a progressive disease in which the muscles do not function properly) in adults and children 2 years of age and older. Deflazacort is in a class of medications called corticosteroids. It works by reducing inflammation (swelling) and by changing the way the immune system works. (Sourced from: https://medlineplus.gov/druginfo/meds/a617024.html)
Mechanisms of Action: GR Agonist
Novel Mechanism: No
Modality: Small Molecule
Route of Administration: Oral
FDA Designation: None *
Approval Status: Approved
Approved Countries: Argentina | Bangladesh | Brazil | Chile | Colombia | Czech | Denmark | Dominican Republic | Ecuador | Egypt | Finland | Germany | Greece | India | Ireland | Italy | Jordan | Korea | Lebanon | Lithuania | Luxembourg | Mexico | Morocco | Netherlands | New Zealand | Norway | Pakistan | Peru | Philippines | Portugal | Russia | Serbia | Slovenia | Spain | Sri Lanka | Sweden | Switzerland | Turkey | United Arab Emirates | United Kingdom | United States | Uruguay | Venezuela | Vietnam
Approved Indications: None
Known Adverse Events: None
Company: PTC Therapeutics
Company Location: SOUTH PLAINFIELD NJ 07080-2449
Company CEO: Stuart W. Peltz
Additonal Commercial Interests: None
Clinical Description
Countries in Clinic:
Active Clinical Trial Count:
Highest Development Phases
Phase 4: Kidney Failure, Chronic|Tooth, Impacted|Trismus|Pain, Postoperative|Dermatitis
Phase 3: Muscular Dystrophies, Limb-Girdle|Muscular Dystrophy, Duchenne|Pulmonary Aspergillosis|Aspergillosis, Allergic Bronchopulmonary
Phase 2: Muscular Dystrophies
Phase 1: Hepatic Insufficiency|Muscular Dystrophy, Duchenne|Healthy Volunteers|Kidney Diseases|Arthritis, Rheumatoid
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
|---|---|---|---|---|---|
| RA/2020/001 | P3 |
Active, not recruiting |
Aspergillosis, Allergic Bronchopulmonary|Pulmonary Aspergillosis |
2025-07-01 |
|
| TREAT LMNA | P2 |
Unknown Status |
Muscular Dystrophies |
2025-03-06 |
|
| RACC | N/A |
Recruiting |
Proteinuria|IgA Nephropathy|Kidney Diseases|Glomerulonephritis |
2024-12-01 |
|
| NCT04365088 | P4 |
Active, not recruiting |
Tooth, Impacted|Pain, Postoperative|Trismus |
2023-11-15 |