Deflazacort

Summary

Deflazacort (CHEMBL1201891) is an approved small molecule (ATC H02AB13); indicated across 7 conditions including duchenne muscular dystrophy and muscular dystrophy.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: H02AB13
• Indications: 7 conditions
• Clinical trials: 13
• Chemistry: 441.5 Da · C25H31NO6

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL1201891
Name	Deflazacort
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	189821
ATC	H02AB13
Molecular formula	C25H31NO6
Molecular weight	441.5
InChIKey	FBHSPRKOSMHSIF-GRMWVWQJSA-N

SMILES: CC1=N[C@@]2([C@H](O1)C[C@@H]3[C@@]2(C[C@@H]([C@H]4[C@H]3CCC5=CC(=O)C=C[C@]45C)O)C)C(=O)COC(=O)C

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

Also known as: Azacortinol, Calcort, Deflazacort, DL-458IT, Emflaza, L-5458, MDL 458, MDL-458, MDL458, Zamene, DEFLAZACORT, SID144206059

Patent coverage: 3,547 distinct patent families (14,339 SureChEMBL compound mentions), from 1 matched compound structure(s). Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.

Targets

Targets

Broader ChEMBL bioactivity targets: 2 (assay-derived). Sample: Glucocorticoid receptor, Progesterone receptor.

Bioactivity

ChEMBL activities: 2 potent at pChembl ≥ 5 of 2 total. Top 100 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
NR3C1	6.18	AC50	660	nM	CHEMBL_ACT_25176115
PGR	5.43	AC50	3700	nM	CHEMBL_ACT_25223264

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

2 approved indications. FDA phase 4, plus an anticancer drug’s labelled cancer uses (which ChEMBL often logs at phase 3).

Indication	Phase	MONDO	EFO
Duchenne muscular dystrophy	4	MONDO:0010679	MONDO:0010679
muscular dystrophy	4	MONDO:0020121	MONDO:0020121

4 diseases in clinical trials (phase 1–3, investigational — not approved indications). Highest ChEMBL trial phase per disease; a non-cancer approved use is occasionally logged at phase 3 here.

Disease (in trials)	Phase	MONDO	EFO
limb-girdle muscular dystrophy	3	MONDO:0016971	MONDO:0016971
allergic bronchopulmonary aspergillosis	2	MONDO:0015243	EFO:0007140
liver disorder	1	MONDO:0005154	EFO:0001421
kidney disorder	1	MONDO:0005240	EFO:0003086

1 further indication record had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 13.

Phase distribution

Phase	Trials
PHASE1	7
PHASE3	3
PHASE2/PHASE3	2
Not specified	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT04227483	PHASE2/PHASE3	ACTIVE_NOT_RECRUITING	Deflazacort Vs. Prednisolone in Acute-stage ABPA
NCT00527228	PHASE2/PHASE3	COMPLETED	Deflazacort in Dysferlinopathies
NCT01603407	PHASE3	COMPLETED	Finding the Optimum Regimen for Duchenne Muscular Dystrophy
NCT03642145	PHASE3	WITHDRAWN	A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD)
NCT03783923	PHASE3	TERMINATED	A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)
NCT02251600	PHASE1	COMPLETED	A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy
NCT02286609	PHASE1	COMPLETED	A Single Dose Evaluation of the Effects of Moderate Hepatic Impairment on Deflazacort Pharmacokinetics
NCT02286622	PHASE1	COMPLETED	A Single Dose Evaluation of the Effects of Renal Impairment on Deflazacort Pharmacokinetics
NCT02286635	PHASE1	COMPLETED	Evaluate Effects of Multiple Doses of Rifampin and Clarithromycin on the Single Dose Pharmacokinetics of Deflazacort
NCT02295748	PHASE1	COMPLETED	An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability Deflazacort
NCT02485431	PHASE1	COMPLETED	Food Effect and Bioavailability of Deflazacort Formulations in Healthy Volunteers
NCT04791345	PHASE1	UNKNOWN	Study of the Excretion of Orally Administered Corticosteroids for the Improval of the Detection of Said Substances in Anti-doping Controls
NCT02592941	Not specified	APPROVED_FOR_MARKETING	Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No PharmGKB pharmacogenomic data curated for this drug.

Related molecules

Related molecules

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).