Deflazacort

DB11921ApprovedSmall moleculeCorticosteroids

Deflazacort is a corticosteroid used to treat Duchenne muscular dystrophy. It is marketed by Marathon Pharmaceuticals and was approved in February 2017 by the FDA.

Mechanism: Deflazacort is a corticosteroid prodrug with an active metabolite, 21-deflazacort, which binds to the glucocorticoid receptor to exert anti-inflammatory and immunosuppressive effects on the body. The exact mechanism by which deflazacort exerts its therapeutic effects in patients with DMD is unknown but likely occurs via its anti-inflammatory activities.
Curator reviewed · 4 references
Primary indication: Deflazacort is indicated for the treatment of Duchenne Muscular Dystrophy (DMD) in patients 2 years of age and older.Curator reviewed · 1 structured indication
Formula / weight: C₂₅H₃₁NO₆ · 441.524 g/mol (avg)
First approval: United States, 2017
Also known as: 11.BETA.,21-DIHYDROXY-2'-METHYL-5'.BETA.H-PREGNA-1,4-DIENO(17,16-D)OXAZOLE-3,20-DIONE 21-ACETATE · 5'H-PREGNA-1,4-DIENO(17,16-D)OXAZOLE-3,20-DIONE, 21-(ACETYLOXY)-11-HYDROXY-2'-METHYL-, (11.BETA.,16.BETA.)-
Code names: MDL-458
Brand names: Emflaza
Jaythari
Kymbee
Pyquvi

Resolves to

Compound

UNIIKR5YZ6AE4BCAS14484-47-0ChEMBLCHEMBL1201891ChEBI135720PubChem189821KEGGD03671

Clinical / RWD

RxNorm22396ATCH02AB13

Structure

InChIKeyFBHSPRKOSMHSIF-GRMWVWQJSA-NSMILES[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

Targets

P04150NR3C1

What you can answer from here — as of September 23, 2026

1Protein targetEach mapped to UniProt, with action and pharmacological action

Listed above

1,149Drug interactionsStructured to mechanism, not free text

21Clinical trialsPhase, status and sponsor resolved per trial

1Structured indicationCondition, population, route and combination as fields, not prose

1ContraindicationEach with its own population and attribute set

122Marketed productsAcross 5 countries and 35 labellers

1ATC codeIncluding every combination product, plus 21 drug categories

25+ReferencesStructured and connected to the statements they support

Which drugs share a target with Deflazacort, and which of those have an active Phase 3 trial?

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