Deferiprone

Summary

Deferiprone (CHEMBL70927) is an approved small-molecule iron chelator (ATC V03AC02); indicated across 15 conditions including thalassemia and sickle cell disease.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: V03AC02
• Indications: 15 conditions
• Clinical trials: 51
• Chemistry: 139.15 Da · C7H9NO2

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL70927
Name	Deferiprone
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	2972
ChEBI	CHEBI:68554
ATC	V03AC02
Molecular formula	C7H9NO2
Molecular weight	139.15
InChIKey	TZXKOCQBRNJULO-UHFFFAOYSA-N

SMILES: CC1=C(C(=O)C=CN1C)O

IUPAC name: 3-hydroxy-1,2-dimethylpyridin-4-one

ChEBI definition: A member of the class of 4-pyridones that is pyridin-4(1H)-one substituted at positions 1 and 2 by methyl groups and at position 3 by a hydroxy group. A lipid-soluble iron-chelator used for treatment of thalassaemia.

Pharmacological roles (ChEBI): iron chelator, protective agent.

Also known as: APO-066, APO-66, CP-20, CP20, Deferipron, Deferiprona, Deferiprone, Deferiprone lipomed, DN-18001AF, Ferriprox, L-1, L1

Patent coverage: 2,929 distinct patent families (7,561 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: 12 (assay-derived). Sample: Lysine-specific demethylase 4E, Fructose-bisphosphate aldolase, 4’-phosphopantetheinyl transferase ffp, Lysine-specific demethylase 2A, Lysine-specific demethylase 6A, Lysine-specific demethylase 5C, Cytochrome P450 2C9, Cytochrome P450 2C19, Lysine-specific demethylase 2B, Deoxyhypusine hydroxylase.

Bioactivity

ChEMBL activities: 14 potent at pChembl ≥ 5 of 17 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
Q9F4F7	5.85	Potency	1412	nM	CHEMBL_ACT_4384748
CYP2C9	5.6	Potency	2512	nM	CHEMBL_ACT_5065453
CYP2C19	5.5	Potency	3162	nM	CHEMBL_ACT_4014342
KDM4A	5.49	IC50	3220	nM	CHEMBL_ACT_24824655
KDM4A	5.48	IC50	3330	nM	CHEMBL_ACT_24824658
KDM6A	5.38	IC50	4200	nM	CHEMBL_ACT_24775864
A8B2U2	5.35	Potency	4456	nM	CHEMBL_ACT_4609575
DOHH	5.3	IC50	5000	nM	CHEMBL_ACT_19246880
KDM5C	5.25	IC50	5600	nM	CHEMBL_ACT_24775863
KDM2A	5.17	IC50	6800	nM	CHEMBL_ACT_24775861
KDM4E	5.1	Potency	7943	nM	CHEMBL_ACT_3742073
A8B2U2	5.1	Potency	7924	nM	CHEMBL_ACT_4610692
KDM2B	5.09	IC50	8100	nM	CHEMBL_ACT_24775862
KDM4E	5.05	Potency	8912	nM	CHEMBL_ACT_3718548

Target pathways

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

Indications & clinical

Indications

15 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
thalassemia	4	MONDO:0000984	EFO:1001996
sickle cell disease	4	MONDO:0011382	MONDO:0011382
beta thalassemia	4	MONDO:0019402	MONDO:0016486
acute kidney injury	3	MONDO:0002492	HP:0001919
pantothenate kinase-associated neurodegeneration	3	MONDO:0009319	MONDO:0009319
stroke disorder	2	MONDO:0005098	EFO:0000712
amyotrophic lateral sclerosis	2	MONDO:0004976	MONDO:0004976
Parkinson disease	2	MONDO:0005180	MONDO:0005180
Friedreich ataxia	2	MONDO:0100339	MONDO:0100339
HIV infectious disease	1	MONDO:0005109	EFO:0000764
acute myocardial infarction	1	MONDO:0004781	EFO:0008583
cerebellar ataxia	0	MONDO:0000437	MONDO:0011426

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

Clinical trials

Total trials: 51.

Phase distribution

Phase	Trials
PHASE2	14
PHASE4	9
PHASE3	7
PHASE1	6
PHASE2/PHASE3	5
Not specified	5
PHASE1/PHASE2	3
EARLY_PHASE1	2

Top trials by phase / activity

NCT	Phase	Status	Title
NCT00103753	PHASE4	UNKNOWN	Combined Chelation Treatment With Deferiprone and Deferoxamine in Thalassemia Major
NCT00105495	PHASE4	COMPLETED	Efficacy Study in Removing Excess Iron From the Heart
NCT00733811	PHASE4	COMPLETED	Efficacy Study of the Use of Sequential DFP-DFO Versus DFP
NCT00800761	PHASE4	COMPLETED	Intensive Combined Chelation Therapy for Iron-Induced Cardiac Disease in Patients With Thalassemia Major
NCT01770652	PHASE4	COMPLETED	An Open-label, Non-randomized, Parallel Group Study in Subjects With Mild, Moderate, Severe, or No Renal Impairment
NCT01860703	PHASE4	COMPLETED	Evaluation of Whether Deferiprone Affects QT Interval in Healthy Subjects
NCT02041299	PHASE4	TERMINATED	Efficacy and Safety of Ferriprox® in Patients With Sickle Cell Disease or Other Anemias
NCT02443545	PHASE4	TERMINATED	Long-term Safety and Efficacy of Ferriprox® in Iron Overloaded Patients With Sickle Cell Disease or Other Anemias
NCT03591575	PHASE4	COMPLETED	Safety and Efficacy of Early Treatment With Deferiprone in Infants and Young Children
NCT07092930	PHASE3	RECRUITING	Effects of β-alanine and Sodium Bicarbonate Supplementation on Physical Capacity and Biochemical Markers Concentrations
NCT00350662	PHASE3	COMPLETED	Study With Deferiprone and/or Desferrioxamine in Iron Overloaded Patients
NCT00529152	PHASE3	COMPLETED	Safety and Efficacy of Ferriprox™ (Deferiprone) Oral Solution in Iron Overloaded Pediatric Patients
NCT00943748	PHASE2/PHASE3	COMPLETED	Efficacy and Safety of the Iron Chelator Deferiprone in Parkinson’s Disease
NCT01391520	PHASE3	WITHDRAWN	Efficacy and Safety of Deferiprone in Patients With Chronic Kidney Disease Undergoing a Cardiac Catheterization and Receiving Contrast Agent
NCT01741532	PHASE3	COMPLETED	Efficacy and Safety Study of Deferiprone in Patients With Pantothenate Kinase-associated Neurodegeneration (PKAN)
NCT01825512	PHASE3	COMPLETED	Efficacy/Safety Study of Deferiprone Compared to Deferasirox in Paediatric Patients
NCT02083575	PHASE2/PHASE3	UNKNOWN	Role of Vitamin C to Augment Iron Chelation With DFP or DFX
NCT02173951	PHASE2/PHASE3	UNKNOWN	An Algorithm to Start Iron Chelation in Minimally Transfused Young Beta-thalassemia Major Patients
NCT02174848	PHASE3	COMPLETED	Long-term Deferiprone Treatment in Patients With Pantothenate Kinase-Associated Neurodegeneration
NCT02882477	PHASE2/PHASE3	UNKNOWN	Treatment of Wolfram Syndrome Type 2 With the Chelator Deferiprone and Incretin Based Therapy
NCT03293069	PHASE2/PHASE3	COMPLETED	Conservative Iron Chelation as a Disease-modifying Strategy in Amyotrophic Lateral Sclerosis
NCT03754725	PHASE1/PHASE2	RECRUITING	Ferritin and Iron Burden in SAH sIRB
NCT05604131	PHASE2	RECRUITING	Cardiac MRI-guided Deferiprone Therapy for Acute Myocardial Infarction Patients
NCT07023666	PHASE2	RECRUITING	Early Screening and Treatment of Heart Complication in Sickle Cell Disease
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
NCT00530127	PHASE1/PHASE2	COMPLETED	A Study Investigating the Safety and Tolerability of Deferiprone in Patients With Friedreich’s Ataxia
NCT00897221	PHASE2	COMPLETED	A Study Investigating the Long-term Safety and Efficacy of Deferiprone in Patients With Friedreich’s Ataxia
NCT00907283	PHASE2	UNKNOWN	Ferrochelating Treatment in Patients Affected by Neurodegeneration With Brain Iron Accumulation (NBIA)
NCT01539837	PHASE2	COMPLETED	A Pilot Clinical Trial With the Iron Chelator Deferiprone in Parkinson’s Disease
NCT01709032	PHASE1/PHASE2	COMPLETED	Combination Deferasirox and Deferiprone for Severe Iron Overload in Thalassemia
NCT01740713	PHASE2	COMPLETED	Pharmacokinetic Study of Deferiprone in Paediatric Patients
NCT02164253	PHASE2	COMPLETED	Focal Accumulation of Iron in Cerebral Regions in Early ALS (Amyotrophic Lateral Sclerosis) Patients
NCT02477631	PHASE2	COMPLETED	Effect of Deferiprone on Oxidative-Stress and Iron-Overload in Low Risk Transfusion-Dependent MDS Patients
NCT02655315	PHASE2	COMPLETED	Conservative Iron Chelation as a Disease-modifying Strategy in Parkinson’s Disease
NCT02728843	PHASE2	COMPLETED	Study of Parkinson’s Early Stage With Deferiprone
NCT04000438	PHASE2	COMPLETED	Effect of Tafoxiparin on Cervical Ripening and Induction of Labor in Term Pregnant Women With an Unripe Cervix
NCT05111821	PHASE2	TERMINATED	Iron Chelation in the Prevention of Secondary Degeneration After Stroke
NCT01989455	PHASE1	COMPLETED	A Blinded, Placebo-Controlled Study of the Safety and Pharmacokinetics of Single Doses of Intravenous Deferiprone in Healthy Volunteers
NCT02189941	PHASE1	COMPLETED	Pilot Study of the Pharmacokinetic Profile of Deferiprone Sustained-Release Formulation in Healthy Volunteers

Clinical evidence (CIViC)

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

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline, but PharmGKB curates 1 clinical and 8 variant annotation(s) for this drug (gene-keyed; see PharmGKB).

Related molecules

Related molecules

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