Deferasirox

Summary

Deferasirox (CHEMBL550348) is an approved small-molecule iron chelator (ATC V03AC03); indicated across 17 conditions including beta thalassemia and myelodysplastic syndrome.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: V03AC03
• Indications: 17 conditions
• Clinical trials: 82
• Chemistry: 373.4 Da · C21H15N3O4

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL550348
Name	Deferasirox
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	214348
ChEBI	CHEBI:49005
ATC	V03AC03
Molecular formula	C21H15N3O4
Molecular weight	373.4
InChIKey	BOFQWVMAQOTZIW-UHFFFAOYSA-N

SMILES: C1=CC=C(C(=C1)C2=NN(C(=N2)C3=CC=CC=C3O)C4=CC=C(C=C4)C(=O)O)O

IUPAC name: 4-[3,5-bis(2-hydroxyphenyl)-1,2,4-triazol-1-yl]benzoic acid

ChEBI definition: A member of the class of triazoles, deferasirox is 1,2,4-triazole substituted by a 4-carboxyphenyl group at position 1 and by 2-hydroxyphenyl groups at positions 3 and 5. An orally active iron chelator, it is used to manage chronic iron overload in patients receiving long-term blood transfusions.

Pharmacological roles (ChEBI): iron chelator.

Also known as: Deferasirox, Deferasirox accord, Deferasirox mylan, Exjade, ICL-670, ICL-670A, ICL670, ICL670A, Jadenu, Jadenu sprinkle, Osveral, deferasirox

Patent coverage: 474 distinct patent families (1,593 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 1,552 (97%) of the total. 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: 8 (assay-derived). Sample: ATP-binding cassette sub-family C member 4, Lysine-specific demethylase 6B, A-type voltage-gated potassium channel KCND3, Sodium channel protein type 5 subunit alpha, Lysine-specific demethylase 5A, Flavin reductase (NADPH), Lysine-specific demethylase 4A, Bile salt export pump.

Bioactivity

ChEMBL activities: 7 potent at pChembl ≥ 5 of 12 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
BLVRB	5.77	Kd	1710	nM	CHEMBL_ACT_24380461
BLVRB	5.77	Kd	1698	nM	CHEMBL_ACT_24380462
KDM4A	5.49	IC50	3220	nM	CHEMBL_ACT_24824656
KDM4A	5.48	IC50	3330	nM	CHEMBL_ACT_24824659
KDM6B	5.4	IC50	3950	nM	CHEMBL_ACT_24775867
KDM4A	5.32	IC50	4760	nM	CHEMBL_ACT_24775865
KDM5A	5.3	IC50	5000	nM	CHEMBL_ACT_24775866

Target pathways

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

Indications & clinical

Indications

17 indications (5 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	4	MONDO:0019402	Orphanet:848
myelodysplastic syndrome	4	MONDO:0018881	EFO:0000198
thalassemia	3	MONDO:0000984	EFO:1001996
hemosiderosis	3	MONDO:0001436	MONDO:0001436
sickle cell disease	3	MONDO:0011382	MONDO:0011382
porphyria cutanea tarda	3	MONDO:0015104	MONDO:0015104
Diamond-Blackfan anemia	3	MONDO:0015253	MONDO:0015253
anemia	2	MONDO:0002280	EFO:0004272
acute myeloid leukemia	2	MONDO:0018874	EFO:0000222
postmenopausal osteoporosis	2	MONDO:0008159	EFO:0003854
Zygomycosis	2	MONDO:0019136	EFO:0007380
hereditary hemochromatosis	2	MONDO:0006507	MONDO:0006507
liver disorder	1	MONDO:0005154	EFO:0001421
acute lymphoblastic leukemia	1	MONDO:0004967	EFO:0000220

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

Clinical trials

Total trials: 82.

Phase distribution

Phase	Trials
PHASE2	35
PHASE4	17
PHASE3	10
Not specified	10
PHASE1	5
PHASE1/PHASE2	4
PHASE2/PHASE3	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT00117507	PHASE4	COMPLETED	Study for the Treatment of Transfusional Iron Overload in Myelodysplastic Patients
NCT00171301	PHASE4	COMPLETED	Extension Study of the Efficacy and Safety of Deferasirox Treatment in Beta-thalassemia Patients With Transfusional Hemosiderosis (Study Amended to 2-year Duration)
NCT00452660	PHASE4	COMPLETED	Evaluation the Effect of Exjade on Oxidative Stress in Low Risk Myelodysplastic Syndrome Patients With Iron Over Load
NCT00481143	PHASE4	COMPLETED	Efficacy and Safety of Deferasirox in Patients With Myelodysplastic Syndrome and Transfusion-dependent Iron Overload
NCT00564941	PHASE4	COMPLETED	Evaluating the Efficacy of Deferasirox in Transfusion Dependent Chronic Anaemias (Myelodysplastic Syndrome, Beta-thalassaemia Patients) With Chronic Iron Overload
NCT00654589	PHASE4	COMPLETED	Efficacy and Safety of Oral Deferasirox (20 mg/kg/d) in Pts 3 to 6 Months After Allogeneic Hematopoietic Cell Transplantation Who Present With Iron Overload
NCT00673608	PHASE4	COMPLETED	Magnetic Resonance Imaging (MRI) Assessments of the Heart and Liver Iron Load in Patients With Transfusion Induced Iron Overload
NCT00749515	PHASE4	COMPLETED	Pilot Study for Patients With Poor Response to Deferasirox
NCT01250951	PHASE4	COMPLETED	This Study Will Evaluate Efficacy and Safety of Deferasirox in Patients With Myelodysplastic Syndromes (MDS), Thalassemia and Rare Anemia Types Having Transfusion-induced Iron Overload.
NCT01326845	PHASE4	TERMINATED	Myelodysplastic Syndrome (MDS) Gastrointestinal (GI) Tolerability Study
NCT01335035	PHASE4	COMPLETED	Open-Label Single-Arm Pilot Study in Adult Allogeneic Hematopoietic Stem Cell Transplant Recipients With Transfusional Iron Overload
NCT01610297	PHASE4	COMPLETED	Post Hematopoietic Stem Cell Transplantation
NCT01709838	PHASE4	COMPLETED	Efficacy and Safety Study of Deferasirox in Patients With Non-transfusion Dependent Thalassemia
NCT01724138	PHASE4	WITHDRAWN	An Open Label Study to Evaluate the Pharmacokinetics, Safety, Tolerability and Efficacy of Deferasirox Administered to Chinese Patients With β-thalassemia Major Aged From 2 to Less Than 6 Years Old
NCT01818726	PHASE4	TERMINATED	Safety and Efficacy of Exjade in the Treatment of Transfusion-dependent Iron Overload in Aplastic Anemia Patients
NCT02069886	PHASE4	WITHDRAWN	Effect of Deferasirox on Endocrine Complications in Subjects With Transfusion Dependent Thalassemia
NCT03372083	PHASE4	COMPLETED	Safety Study of Crushed Deferasirox Film Coated Tablets in Pediatric Patients With Transfusional Hemosiderosis
NCT00061750	PHASE3	COMPLETED	Safety & Efficacy of ICL670 vs. Deferoxamine in Beta-thalassemia Patients With Iron Overload Due to Blood Transfusions
NCT00171171	PHASE3	COMPLETED	A Study of Long-term Treatment With Deferasirox in Patients With Beta-thalassemia and Transfusional Hemosiderosis
NCT00171210	PHASE3	COMPLETED	An Extension Study of Iron Chelation Therapy With Deferasirox (ICL670) in β-thalassemia Patients With Transfusional Iron Overload
NCT00171821	PHASE3	COMPLETED	A Study Assessing the Efficacy and Safety of Deferasirox in Patients With Transfusion-dependent Iron Overload
NCT00235391	PHASE3	COMPLETED	Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload
NCT00469560	PHASE3	COMPLETED	Safety, Tolerability, and Efficacy of Deferasirox in MDS
NCT00599326	PHASE3	COMPLETED	Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda
NCT00981370	PHASE3	TERMINATED	Clinical Importance of Treating Iron Overload in Sickle Cell Disease
NCT01033747	PHASE2/PHASE3	COMPLETED	Safety and Efficacy of Deferasirox in Patients With Transfusion Dependent Iron Overload - a Non-comparative Extension Study
NCT01825512	PHASE3	COMPLETED	Efficacy/Safety Study of Deferiprone Compared to Deferasirox in Paediatric Patients
NCT02720536	PHASE3	COMPLETED	Extended Evaluation of Deferasirox Film-coated Tablet (FCT) Formulation
NCT07023666	PHASE2	RECRUITING	Early Screening and Treatment of Heart Complication in Sickle Cell Disease
NCT00061763	PHASE2	COMPLETED	Study of Deferasirox in Iron Overload From Beta-thalassemia Unable to be Treated With Deferoxamine or Chronic Anemias
NCT00067080	PHASE2	COMPLETED	Safety of ICL670 vs. Deferoxamine in Sickle Cell Disease Patients With Iron Overload Due to Blood Transfusions
NCT00110266	PHASE2	COMPLETED	Study of Deferasirox for Treatment of Transfusional Iron Overload in Myelodysplastic Patients
NCT00110617	PHASE2	COMPLETED	Study of Deferasirox Relative to Subcutaneous Deferoxamine in Sickle Cell Disease Patients
NCT00303329	PHASE2	COMPLETED	Extension Study of Iron Chelation Therapy With Deferasirox in β-thalassemia and Rare Chronic Anemia Patients
NCT00379483	PHASE2	COMPLETED	Extension Study of Iron Chelation Therapy With Deferasirox in Patients With Transfusional Iron Overload
NCT00390858	PHASE2	COMPLETED	A 4-year Extension Study to Core 1-year Study of Iron Chelation Therapy With Deferasirox in β-thalassemia Major Pediatric Patients With Transfusional Iron Overload.
NCT00395629	PHASE1/PHASE2	COMPLETED	Safety and Efficacy of Deferasirox (ICL670) in Patients With Iron Overload Resulting From Hereditary Hemochromatosis
NCT00419770	PHASE2	COMPLETED	The Deferasirox-AmBisome Therapy for Mucormycosis (DEFEAT Mucor) Study
NCT00447694	PHASE2	COMPLETED	Cardiac T2* in Beta-thalassemia Patients on Deferasirox Treatment
NCT00600938	PHASE2	COMPLETED	Evaluating Use of Deferasirox as Compared to Deferoxamine in Treating Cardiac Iron Overload

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).