| Drug Name |
Deflazacort |
| Drug ID |
BADD_D02492 |
| Description |
Deflazacort, also known as Emflaza, is a corticosteroid prodrug used as an agent to manage Duchenne Muscular Dystrophy (DMD). It is marketed by Marathon Pharmaceuticals and was approved in February 2017 by the FDA.[L6694,FDA label]
Duchenne Muscular Dystrophy is an inherited disorder resulting from mutations of the dystrophin gene, which is important for muscle function. This disease can cause serious muscle weakness and progressive breathing and cardiovascular disability, severely impacting patient quality of life and survival.[A179446,A179449,L6697] This disease usually manifests by muscle weakness in early childhood followed by loss of the ability to walk (ambulation) as early as age 7.[A179449]
Deflazacort delays the onset of muscle related complications resulting from DMD[A179455], prolonging the lives of children diagnosed with this disease and exerting less harmful effects on the bone health and weight than other steroid medications.[A179452,A25340] |
| Indications and Usage |
Deflazacort is indicated for the treatment of Duchenne Muscular Dystrophy (DMD) in patients 2 years of age and older.[FDA label] |
| Marketing Status |
approved; investigational |
| ATC Code |
H02AB13 |
| DrugBank ID |
DB11921
|
| KEGG ID |
D03671
|
| MeSH ID |
C021988
|
| PubChem ID |
189821
|
| TTD Drug ID |
D09WYX
|
| NDC Product Code |
52128-135; 24002-0036; 52856-503; 52856-501; 64918-1111; 57582-040; 64918-1901; 52856-502; 52856-504; 22552-0027; 52856-505 |
| UNII |
KR5YZ6AE4B
|
| Synonyms |
deflazacort | Dezacor | Emflaza | Calcort | Zamene |
