Deferoxamine

Summary

Deferoxamine (CHEMBL556) is an approved small-molecule iron chelator (ATC V03AC01); indicated across 12 conditions including beta thalassemia and hereditary hemochromatosis.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: V03AC01
• Indications: 12 conditions
• Clinical trials: 45
• Chemistry: 560.7 Da · C25H48N6O8

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL556
Name	Deferoxamine
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	2973
ChEBI	CHEBI:4356
ATC	V03AC01
Molecular formula	C25H48N6O8
Molecular weight	560.7
InChIKey	UBQYURCVBFRUQT-UHFFFAOYSA-N

SMILES: CC(=O)N(CCCCCNC(=O)CCC(=O)N(CCCCCNC(=O)CCC(=O)N(CCCCCN)O)O)O

IUPAC name: N-[5-[[4-[5-[acetyl(hydroxy)amino]pentylamino]-4-oxobutanoyl]-hydroxyamino]pentyl]-N’-(5-aminopentyl)-N’-hydroxybutanediamide

ChEBI definition: An acyclic desferrioxamine that is butanedioic acid in which one of the carboxy groups undergoes formal condensation with the primary amino group of N-(5-aminopentyl)-N-hydroxyacetamide and the second carboxy group undergoes formal condensation with the hydroxyamino group of N1-(5-aminopentyl)-N1-hydroxy-N4-[5-(hydroxyamino)pentyl]butanediamide. It is a siderophore native to Streptomyces pilosus biosynthesised by the DesABCD enzyme cluster as a high affinity Fe(III) chelator.

Pharmacological roles (ChEBI): iron chelator, siderophore, ferroptosis inhibitor.

Other ChEBI roles (chemical / environmental): bacterial metabolite.

Also known as: Deferoxamina, Deferoxamine, Desferoxamine b, Desferrioxamine, Desferrioxamine b, Dfo-b, NSC-527604, desferrioxamine, deferoxamine B, Desferrioxamine B, desferal, Desferal

Parent form; salt/anhydrous children: CHEMBL1234, CHEMBL2002581

Patent coverage: 7,274 distinct patent families (25,751 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: 4 (assay-derived). Sample: Hypoxia-inducible factor 1-alpha, Methylcytosine dioxygenase TET2, Deoxyhypusine hydroxylase, Lysine-specific demethylase 4A.

Bioactivity

ChEMBL activities: 3 potent at pChembl ≥ 5 of 5 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
KDM4A	5.49	IC50	3220	nM	CHEMBL_ACT_24824654
KDM4A	5.48	IC50	3330	nM	CHEMBL_ACT_24824657
DOHH	5.3	IC50	5000	nM	CHEMBL_ACT_19246882

Target pathways

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

Indications & clinical

Indications

12 indications (0 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).

Indication	Trial phase	MONDO	EFO
beta thalassemia	3	MONDO:0019402	Orphanet:848
hereditary hemochromatosis	3	MONDO:0006507	EFO:1000642
severe acute respiratory syndrome	3	MONDO:0005091	MONDO:0100096
intracerebral hemorrhage	2	MONDO:0013792	EFO:0005669
subarachnoid hemorrhage	2	MONDO:0005099	EFO:0000713
breast neoplasm	2	MONDO:0021100	MONDO:0007254
sickle cell disease	2	MONDO:0011382	MONDO:0011382
thalassemia	1	MONDO:0000984	EFO:1001996
hepatocellular carcinoma	1	MONDO:0007256	EFO:0000182

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

Clinical trials

Total trials: 45.

Phase distribution

Phase	Trials
PHASE2	17
PHASE1/PHASE2	6
Not specified	6
PHASE4	4
PHASE3	4
PHASE1	4
PHASE2/PHASE3	2
EARLY_PHASE1	2

Top trials by phase / activity

NCT	Phase	Status	Title
NCT00105495	PHASE4	COMPLETED	Efficacy Study in Removing Excess Iron From the Heart
NCT00749515	PHASE4	COMPLETED	Pilot Study for Patients With Poor Response to Deferasirox
NCT00800761	PHASE4	COMPLETED	Intensive Combined Chelation Therapy for Iron-Induced Cardiac Disease in Patients With Thalassemia Major
NCT02041299	PHASE4	TERMINATED	Efficacy and Safety of Ferriprox® in Patients With Sickle Cell Disease or Other Anemias
NCT06763055	PHASE3	RECRUITING	The Fifth INTEnsive pReventing Secondary Injury in Acute Cerebral Haemorrhage Trial Within ACT-GLOBAL
NCT07162363	PHASE2/PHASE3	NOT_YET_RECRUITING	Synergistic Minimally Invasive Surgery and Deferoxamine in ICH
NCT00061750	PHASE3	COMPLETED	Safety & Efficacy of ICL670 vs. Deferoxamine in Beta-thalassemia Patients With Iron Overload Due to Blood Transfusions
NCT00350662	PHASE3	COMPLETED	Study With Deferiprone and/or Desferrioxamine in Iron Overloaded Patients
NCT04292314	PHASE2/PHASE3	COMPLETED	Hydroxy Urea, Omega 3, Nigella Sativa,Honey on Oxidative Stress and Iron Chelation in Pediatric Major Thalassemia
NCT04361032	PHASE3	UNKNOWN	Assessment of Efficacy and Safety of Tocilizumab Compared to DefeROxamine, Associated With Standards Treatments in COVID-19 (+) Patients Hospitalized In Intensive Care in Tunisia
NCT03137966	PHASE2	NOT_YET_RECRUITING	Effect of Deferoxamine on Wound Healing Rate in Patients With Diabetes Foot Ulcers
NCT04566991	PHASE2	RECRUITING	Deferoxamine In the Treatment of Aneurysmal Subarachnoid Hemorrhage (aSAH)
NCT04633889	PHASE2	ACTIVE_NOT_RECRUITING	Deferoxamine for the Prevention of Cardiac Surgery-Associated Acute Kidney Injury
NCT07023666	PHASE2	RECRUITING	Early Screening and Treatment of Heart Complication in Sickle Cell Disease
NCT00000595	PHASE2	COMPLETED	Evaluation of Subcutaneous Desferrioxamine as Treatment for Transfusional Hemochromatosis
NCT00067080	PHASE2	COMPLETED	Safety of ICL670 vs. Deferoxamine in Sickle Cell Disease Patients With Iron Overload Due to Blood Transfusions
NCT00069862	PHASE1/PHASE2	COMPLETED	Iron Balance Study of DFO and GT56-252 in Patients With Transfusional Iron Overload Secondary to Beta-Thalassemia
NCT00110617	PHASE2	COMPLETED	Study of Deferasirox Relative to Subcutaneous Deferoxamine in Sickle Cell Disease Patients
NCT00115349	PHASE2	TERMINATED	Combination Therapy Compared With Single-Drug Therapy in Patients With Cardiac Diseases
NCT00349453	PHASE2	COMPLETED	Study Using Deferiprone Alone or in Combination With Desferrioxamine in Iron Overloaded Transfusion-dependent Patients
NCT00600938	PHASE2	COMPLETED	Evaluating Use of Deferasirox as Compared to Deferoxamine in Treating Cardiac Iron Overload
NCT00738413	PHASE1/PHASE2	UNKNOWN	Iron Balance Study of Deferasirox, Deferoxamine and the Combination of Both
NCT00777140	PHASE2	COMPLETED	Thrombolysis and Deferoxamine in Middle Cerebral Artery Occlusion
NCT00870883	PHASE2	COMPLETED	N-acetylcysteine Plus Deferoxamine for Patients With Hypotension
NCT01254227	PHASE2	COMPLETED	Efficacy and Safety of Deferasirox in Combination With Deferoxamine Followed by Deferasirox Monotherapy in Severe Cardiac Iron Overload
NCT01459718	PHASE2	TERMINATED	Safety and Efficacy of Deferasirox in Combination With Desferoxamine in β-thalassaemia Patients With Severe Cardiac Iron Overload
NCT01662895	PHASE2	TERMINATED	High-Dose Deferoxamine in Intracerebral Hemorrhage
NCT02175225	PHASE2	COMPLETED	Intracerebral Hemorrhage Deferoxamine Trial - iDEF Ttrial
NCT02367248	PHASE1/PHASE2	UNKNOWN	Deferoxamine and Xingnaojing Injection Treatment in Intracerebral Hemorrhage
NCT02875262	PHASE1/PHASE2	UNKNOWN	Deferoxamine in Aneurysmal Subarachnoid Hemorrhage Trial
NCT03085771	PHASE2	UNKNOWN	Desferal Administration to Improve the Impaired Reaction to Hypoxia in Diabetes
NCT04058197	PHASE1/PHASE2	WITHDRAWN	Deferoxamine for Sickle Cell Chronic Leg Ulcer Treatment
NCT04333550	PHASE1/PHASE2	UNKNOWN	Application of Desferal to Treat COVID-19
NCT05184816	PHASE1	RECRUITING	A Study of Deferoxamine (DFO) in People With Leptomeningeal Metastasis
NCT00598572	PHASE1	COMPLETED	Dose Finding and Safety Study of Deferoxamine in Patients With Brain Hemorrhage
NCT01752153	PHASE1	COMPLETED	Immunomodulatory Effects of Silymarin in Patients With Beta-Thalassemia Major
NCT03652467	PHASE1	UNKNOWN	The Safety and Efficacy of Deferoxamine for Treating Unresectable Hepatocellular Carcinoma
NCT01365104	EARLY_PHASE1	COMPLETED	Modulation of Cerebral Blood Flow Using Iron Chelators
NCT02216513	EARLY_PHASE1	TERMINATED	Deferoxamine to Prevent Delayed Cerebral Ischemia After Subarachnoid Hemorrhage
NCT00000623	Not specified	COMPLETED	Thalassemia (Cooley’s Anemia) Clinical Research Network (TCRN)

Clinical evidence (CIViC)

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

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline or drug-level clinical/variant annotations in PharmGKB for this molecule.

Related molecules

Related molecules

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