Basic Information
ID DDInter492
Drug Type small molecule
Molecular Formula C25H31NO6
Molecular Weight 441.524
CAS Number 14484-47-0
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]
ATC Classification H02AB13
IUPAC Name 2-[(1S,2S,4R,8S,9S,11S,12S,13R)-11-hydroxy-6,9,13-trimethyl-16-oxo-5-oxa-7-azapentacyclo[10.8.0.0²,⁹.0⁴,⁸.0¹³,¹⁸]icosa-6,14,17-trien-8-yl]-2-oxoethyl acetate
InChI FBHSPRKOSMHSIF-GRMWVWQJSA-N
Canonical SMILES [H][C@@]12C[C@@]3([H])[C@]4([H])CCC5=CC(=O)C=C[C@]5(C)[C@@]4([H])[C@@]([H])(O)C[C@]3(C)[C@@]1(N=C(C)O2)C(=O)COC(C)=O
Useful Links DrugBank ChEBI PubChem Substance KEGG Drug ChemSpider Wikipedia ChEMBL ZINC
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