DEFERIPRONE

Details

Stereochemistry	ACHIRAL
Molecular Formula	C7H9NO2
Molecular Weight	139.1519
Optical Activity	NONE
Defined Stereocenters	0 / 0
E/Z Centers	0
Charge	0

SHOW SMILES / InChI

Structure Search

SMILES

CN1C=CC(=O)C(O)=C1C

InChI

InChIKey=TZXKOCQBRNJULO-UHFFFAOYSA-N

InChI=1S/C7H9NO2/c1-5-7(10)6(9)3-4-8(5)2/h3-4,10H,1-2H3

HIDE SMILES / InChI

Molecular Formula	C7H9NO2
Molecular Weight	139.1519
Charge	0
Count

Stereochemistry	ACHIRAL
Additional Stereochemistry	No
Defined Stereocenters	0 / 0
E/Z Centers	0
Optical Activity	NONE

Description
Sources: http://www.accessdata.fda.gov/drugsatfda_docs/label/2011/021825lbl.pdf
Curator's Comment: description was created based on several sources, including https://pubchem.ncbi.nlm.nih.gov/compound/deferiprone#section=Top; http://www.ncbi.nlm.nih.gov/pubmed/12825969

Deferiprone (trade name Ferriprox) is an iron chelator indicated for the treatment of patients with transfusional iron overload due to thalassemia syndromes when current chelation therapy is inadequate. Deferiprone is an orally bioavailable bidentate ligand with iron chelating activity. Deferiprone binds to iron in a 3:1 (ligand:iron) molar ratio. By binding to iron, deferiprone is able to remove excess iron from the body. All the adverse effects of deferiprone are considered reversible, controllable and manageable. These include agranulocytosis with frequency of about 0.6%, neutropenia 6%, musculoskeletal and joint pains 15%, gastrointestinal complains 6% and zinc deficiency 1%.

CNS Activity

Originator

Approval Year

Targets

Primary Target	Pharmacology	Condition	Potency
Iron 18 Target ID: CHEMBL2363058 Sources: http://www.accessdata.fda.gov/drugsatfda_docs/label/2011/021825lbl.pdf	Chelating Agent 139

Conditions

Condition	Modality	Targets	Highest Phase	Product
Thalassemia syndrome 1 Sources: http://www.accessdata.fda.gov/drugsatfda_docs/label/2011/021825lbl.pdf	Secondary		Approved 8583	FERRIPROX Approved Use Indicated for the treatment of patients with transfusional iron overload due to thalassemia syndromes when current chelation therapy is inadequate. Approval is based on a reduction in serum ferritin levels. There are no controlled trials demonstrating a direct treatment benefit, such as improvement in disease-related symptoms, functioning, or increased survival Launch Date 2011
Iron overload 4 Sources: http://www.webmd.com/drugs/2/drug-158383/deferiprone-oral/details/list-conditions	Primary		Approved 8583	FERRIPROX Approved Use Indicated for the treatment of patients with transfusional iron overload due to thalassemia syndromes when current chelation therapy is inadequate. Approval is based on a reduction in serum ferritin levels. There are no controlled trials demonstrating a direct treatment benefit, such as improvement in disease-related symptoms, functioning, or increased survival. Safety and effectiveness have not been established for the treatment of transfusional iron overload in patients with other chronic anemias. Launch Date 2011

C_max

Value	Dose	Analyte	Population
33.4 ug/mL Clinical Trial https://clinicaltrials.gov/ct2/show/NCT01770652	33 mg/kg single, oral dose: 33 mg/kg route of administration: oral experiment type: single co-administered:	DEFERIPRONE serum	Homo sapiens
43.3 ug/mL Clinical Trial https://clinicaltrials.gov/ct2/show/NCT01770652	33 mg/kg single, oral dose: 33 mg/kg route of administration: oral experiment type: single co-administered:	DEFERIPRONE serum	Homo sapiens
30.6 ug/mL Clinical Trial https://clinicaltrials.gov/ct2/show/NCT01770652	33 mg/kg single, oral dose: 33 mg/kg route of administration: oral experiment type: single co-administered:	DEFERIPRONE serum	Homo sapiens
60.8 ug/mL Clinical Trial https://clinicaltrials.gov/ct2/show/NCT01770652	33 mg/kg single, oral dose: 33 mg/kg route of administration: oral experiment type: single co-administered:	DEFERIPRONE 3-O-BETA-D-GLUCURONIDE serum	Homo sapiens
118.8 ug/mL Clinical Trial https://clinicaltrials.gov/ct2/show/NCT01770652	33 mg/kg single, oral dose: 33 mg/kg route of administration: oral experiment type: single co-administered:	DEFERIPRONE 3-O-BETA-D-GLUCURONIDE serum	Homo sapiens
150.8 ug/mL Clinical Trial https://clinicaltrials.gov/ct2/show/NCT01770652	33 mg/kg single, oral dose: 33 mg/kg route of administration: oral experiment type: single co-administered:	DEFERIPRONE 3-O-BETA-D-GLUCURONIDE serum	Homo sapiens
34.1 ug/mL Clinical Trial https://clinicaltrials.gov/ct2/show/NCT01860703	33 mg/kg single, oral dose: 33 mg/kg route of administration: oral experiment type: single co-administered:	DEFERIPRONE plasma	Homo sapiens population: healthy age: sex: food status:
35.2 ug/mL Clinical Trial https://clinicaltrials.gov/ct2/show/NCT01860703	33 mg/kg single, oral dose: 33 mg/kg route of administration: oral experiment type: single co-administered:	DEFERIPRONE 3-O-BETA-D-GLUCURONIDE plasma	Homo sapiens population: healthy age: sex: food status:
54.4 ug/mL Clinical Trial https://clinicaltrials.gov/ct2/show/NCT01860703	50 mg/kg single, oral dose: 50 mg/kg route of administration: oral experiment type: single co-administered:	DEFERIPRONE plasma	Homo sapiens population: healthy age: sex: food status:
51.4 ug/mL Clinical Trial https://clinicaltrials.gov/ct2/show/NCT01860703	50 mg/kg single, oral dose: 50 mg/kg route of administration: oral experiment type: single co-administered:	DEFERIPRONE 3-O-BETA-D-GLUCURONIDE plasma	Homo sapiens population: healthy age: sex: food status:
64.8 μM EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/27975237/	25 mg/kg single, oral dose: 25 mg/kg route of administration: Oral experiment type: SINGLE co-administered:	DEFERIPRONE plasma	Homo sapiens population: UNHEALTHY age: ADULT sex: FEMALE / MALE food status: FASTED

AUC

Value	Dose	Analyte	Population
95.4 ug*h/mL Clinical Trial https://clinicaltrials.gov/ct2/show/NCT01860703	33 mg/kg single, oral dose: 33 mg/kg route of administration: oral experiment type: single co-administered:	DEFERIPRONE plasma	Homo sapiens population: healthy age: sex: food status:
205.5 ug*h/mL Clinical Trial https://clinicaltrials.gov/ct2/show/NCT01860703	33 mg/kg single, oral dose: 33 mg/kg route of administration: oral experiment type: single co-administered:	DEFERIPRONE 3-O-BETA-D-GLUCURONIDE plasma	Homo sapiens population: healthy age: sex: food status:
152.1 ug*h/mL Clinical Trial https://clinicaltrials.gov/ct2/show/NCT01860703	50 mg/kg single, oral dose: 50 mg/kg route of administration: oral experiment type: single co-administered:	DEFERIPRONE plasma	Homo sapiens population: healthy age: sex: food status:
331.2 ug*h/mL Clinical Trial https://clinicaltrials.gov/ct2/show/NCT01860703	50 mg/kg single, oral dose: 50 mg/kg route of administration: oral experiment type: single co-administered:	DEFERIPRONE 3-O-BETA-D-GLUCURONIDE plasma	Homo sapiens population: healthy age: sex: food status:
15001 μM × min EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/27975237/	25 mg/kg single, oral dose: 25 mg/kg route of administration: Oral experiment type: SINGLE co-administered:	DEFERIPRONE plasma	Homo sapiens population: UNHEALTHY age: ADULT sex: FEMALE / MALE food status: FASTED

T_1/2

Value	Dose	Analyte	Population
1.77 h Clinical Trial https://clinicaltrials.gov/ct2/show/NCT01770652	33 mg/kg single, oral dose: 33 mg/kg route of administration: oral experiment type: single co-administered:	DEFERIPRONE serum	Homo sapiens
2.03 h Clinical Trial https://clinicaltrials.gov/ct2/show/NCT01770652	33 mg/kg single, oral dose: 33 mg/kg route of administration: oral experiment type: single co-administered:	DEFERIPRONE serum	Homo sapiens
2.2 h Clinical Trial https://clinicaltrials.gov/ct2/show/NCT01770652	33 mg/kg single, oral dose: 33 mg/kg route of administration: oral experiment type: single co-administered:	DEFERIPRONE serum	Homo sapiens
2.58 h Clinical Trial https://clinicaltrials.gov/ct2/show/NCT01770652	33 mg/kg single, oral dose: 33 mg/kg route of administration: oral experiment type: single co-administered:	DEFERIPRONE 3-O-BETA-D-GLUCURONIDE serum	Homo sapiens
2.58 h Clinical Trial https://clinicaltrials.gov/ct2/show/NCT01770652	33 mg/kg single, oral dose: 33 mg/kg route of administration: oral experiment type: single co-administered:	DEFERIPRONE 3-O-BETA-D-GLUCURONIDE serum	Homo sapiens
3.35 h Clinical Trial https://clinicaltrials.gov/ct2/show/NCT01770652	33 mg/kg single, oral dose: 33 mg/kg route of administration: oral experiment type: single co-administered:	DEFERIPRONE 3-O-BETA-D-GLUCURONIDE serum	Homo sapiens
1.8 h Clinical Trial https://clinicaltrials.gov/ct2/show/NCT01860703	33 mg/kg single, oral dose: 33 mg/kg route of administration: oral experiment type: single co-administered:	DEFERIPRONE plasma	Homo sapiens population: healthy age: sex: food status:
2.5 h Clinical Trial https://clinicaltrials.gov/ct2/show/NCT01860703	33 mg/kg single, oral dose: 33 mg/kg route of administration: oral experiment type: single co-administered:	DEFERIPRONE 3-O-BETA-D-GLUCURONIDE plasma	Homo sapiens population: healthy age: sex: food status:
1.8 h Clinical Trial https://clinicaltrials.gov/ct2/show/NCT01860703	50 mg/kg single, oral dose: 50 mg/kg route of administration: oral experiment type: single co-administered:	DEFERIPRONE plasma	Homo sapiens population: healthy age: sex: food status:
2.6 h Clinical Trial https://clinicaltrials.gov/ct2/show/NCT01860703	50 mg/kg single, oral dose: 50 mg/kg route of administration: oral experiment type: single co-administered:	DEFERIPRONE 3-O-BETA-D-GLUCURONIDE plasma	Homo sapiens population: healthy age: sex: food status:
168 min EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/27975237/	25 mg/kg single, oral dose: 25 mg/kg route of administration: Oral experiment type: SINGLE co-administered:	DEFERIPRONE plasma	Homo sapiens population: UNHEALTHY age: ADULT sex: FEMALE / MALE food status: FASTED

Doses

Dose	Population	Adverse events
30 mg/kg 2 times / day multiple, oral Recommended Dose: 30 mg/kg, 2 times / day Route: oral Route: multiple Dose: 30 mg/kg, 2 times / day Sources: https://pubmed.ncbi.nlm.nih.gov/31620867	unhealthy, 29–71 Health Status: unhealthy Age Group: 29–71 Sex: M+F Sources: https://pubmed.ncbi.nlm.nih.gov/31620867	Disc. AE: Neutropenic sepsis, Fatigue... AEs leading to discontinuation/dose reduction: Neutropenic sepsis (30%) Fatigue (10%) Sources: https://pubmed.ncbi.nlm.nih.gov/31620867
100 mg/kg multiple, oral Recommended Dose: 100 mg/kg Route: oral Route: multiple Dose: 100 mg/kg Sources: https://pubmed.ncbi.nlm.nih.gov/32470438	unhealthy Health Status: unhealthy Sex: M+F Sources: https://pubmed.ncbi.nlm.nih.gov/32470438	Disc. AE: Arthralgia, Joint effusion... AEs leading to discontinuation/dose reduction: Arthralgia (non-serious) Joint effusion (non-serious) Nausea (non-serious) Abdominal discomfort (non-serious) Fatigue (non-serious) Joint swelling (non-serious) Epistaxis (non-serious) Upper respiratory tract infections (non-serious) Upper abdominal pain (non-serious) Vomiting (non-serious) Palpitation (non-serious) Sources: https://pubmed.ncbi.nlm.nih.gov/32470438
33 mg/kg 3 times / day multiple, oral Recommended Dose: 33 mg/kg, 3 times / day Route: oral Route: multiple Dose: 33 mg/kg, 3 times / day Sources: https://www.accessdata.fda.gov/drugsatfda_docs/label/2011/021825lbl.pdf	unhealthy Health Status: unhealthy Sources: https://www.accessdata.fda.gov/drugsatfda_docs/label/2011/021825lbl.pdf	Disc. AE: Agranulocytosis, Fetal damage... AEs leading to discontinuation/dose reduction: Agranulocytosis (grade 3-5) Fetal damage Sources: https://www.accessdata.fda.gov/drugsatfda_docs/label/2011/021825lbl.pdf
33 mg/kg 3 times / day multiple, oral Recommended Dose: 33 mg/kg, 3 times / day Route: oral Route: multiple Dose: 33 mg/kg, 3 times / day Sources: https://www.accessdata.fda.gov/drugsatfda_docs/label/2011/021825lbl.pdf	unhealthy Health Status: unhealthy Sources: https://www.accessdata.fda.gov/drugsatfda_docs/label/2011/021825lbl.pdf	Disc. AE: Aspartate aminotransferase increased, Alanine aminotransferase increased... AEs leading to discontinuation/dose reduction: Aspartate aminotransferase increased (0.78%) Alanine aminotransferase increased (0.16%) Sources: https://www.accessdata.fda.gov/drugsatfda_docs/label/2011/021825lbl.pdf
33 mg/kg 3 times / day multiple, oral Recommended Dose: 33 mg/kg, 3 times / day Route: oral Route: multiple Dose: 33 mg/kg, 3 times / day Sources: https://www.accessdata.fda.gov/drugsatfda_docs/label/2011/021825lbl.pdf	unhealthy Health Status: unhealthy Sources: https://www.accessdata.fda.gov/drugsatfda_docs/label/2011/021825lbl.pdf	Disc. AE: Nausea, Vomiting... AEs leading to discontinuation/dose reduction: Nausea (1.6%) Vomiting (1.6%) Abdominal pain (1.6%) Sources: https://www.accessdata.fda.gov/drugsatfda_docs/label/2011/021825lbl.pdf
15 mg/kg 2 times / day steady, oral Dose: 15 mg/kg, 2 times / day Route: oral Route: steady Dose: 15 mg/kg, 2 times / day Sources: https://clinicaltrials.gov/ct2/show/NCT01741532	unhealthy Health Status: unhealthy Sources: https://clinicaltrials.gov/ct2/show/NCT01741532	Other AEs: Neutropenia, Intestinal dilatation... Other AEs: Neutropenia (serious, 5 patients) Intestinal dilatation (serious, 1 patient) Intestinal obstruction (serious, 1 patient) Volvulus (serious, 1 patient) Obstruction (serious, 1 patient) Pyrexia (serious, 1 patient) Bacterial disease carrier (serious, 1 patient) Bronchitis (serious, 2 patients) Device related infection (serious, 1 patient) Pneumonia (serious, 2 patients) Wound infection (serious, 1 patient) Chemical eye injury (serious, 1 patient) Postoperative ileus (serious, 1 patient) Toxicity to various agents (serious, 1 patient) Unintentional medical device removal (serious, 1 patient) Oromandibular dystonia (serious, 1 patient) Device malfunction (serious, 1 patient) Urinary bladder rupture (serious, 1 patient) Choking (serious, 1 patient) Cough (serious, 1 patient) Respiratory disorder (serious, 1 patient) Colectomy (serious, 1 patient) Intestinal anastomosis (serious, 1 patient) Intrathecal pump insertion (serious, 1 patient) Laparotomy (serious, 1 patient) Medical device battery replacement (serious, 2 patients) Tracheostomy (serious, 1 patient) Tracheostomy tube removal (serious, 1 patient) Wound treatment (serious, 1 patient) Thrombosis (serious, 1 patient) Anaemia (below serious, 12 patients) Influenza (below serious, 3 patients) Pain (below serious, 4 patients) Bronchitis (below serious, 7 patients) Nasopharyngitis (below serious, 11 patient) Rhinitis (below serious, 4 patients) Upper respiratory tract infection (below serious, 8 patients) Viral infection (below serious, 4 patients) Laceration (below serious, 6 patients) Body temperature increased (below serious, 3 patients) Neutrophil count decreased (below serious, 10 patients) Serum ferritin decreased (below serious, 19 patients) Iron deficiency (below serious, 9 patients) Arthralgia (below serious, 8 patients) Muscle spasms (below serious, 3 patients) Pain in extremity (below serious, 10 patients) Dystonia (below serious, 25 patients) Migraine (below serious, 3 patients) Urinary incontinence (below serious, 3 patients) Cough (below serious, 10 patients) Oropharyngeal pain (below serious, 9 patients) Rhinorrhoea (below serious, 4 patients) Hyperhidrosis (below serious, 3 patients) Rash (below serious, 4 patients) Sources: https://clinicaltrials.gov/ct2/show/NCT01741532

AEs

Overview

CYP3A4	CYP2C9	CYP2D6	hERG
inhibitor 2252	inhibitor 2438	inhibitor 2507	inhibitor 2217

OverviewOther

Other Inhibitor	Other Substrate	Other Inducer
CYP1A1 132 CYP1A2 2153 CYP2E1 1060	UGT1A6 343 UGT1A7 249 UGT1A8 323 UGT1A9 423 UGT1A10 331 UGT1A1 479 UGT1A3 470 UGT1A4 399 UGT1A5 56 UGT2B4 278 UGT2B7 456 UGT2B10 131 UGT2B15 236 UGT2B17 237 UGT2B28 65

Drug as perpetrator

Target	Modality	Activity
CYP1A1 272	inhibitor 4027 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2011/021825Orig1s000ClinPharmR.pdf#page=68 Page: 68.0	no
CYP1A2 2333	inhibitor 4027 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2011/021825Orig1s000ClinPharmR.pdf#page=68 Page: 68.0	no
CYP2C9 2497	inhibitor 4027 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2011/021825Orig1s000ClinPharmR.pdf#page=68 Page: 68.0	no
CYP2D6 2546	inhibitor 4027 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2011/021825Orig1s000ClinPharmR.pdf#page=68 Page: 68.0	no
CYP2E1 1146	inhibitor 4027 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2011/021825Orig1s000ClinPharmR.pdf#page=68 Page: 68.0	no
CYP3A4 2633	inhibitor 4027 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2011/021825Orig1s000ClinPharmR.pdf#page=68 Page: 68.0	no

Drug as victim

Target	Modality	Activity
UGT1A6 343	substrate 4014 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2011/021825Orig1s000ClinPharmR.pdf#page=18, https://pubmed.ncbi.nlm.nih.gov/18971318/ Page: 18, 26, 27-28, 31, 33, 41	major
UGT1A10 331	substrate 4014 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2011/021825Orig1s000ClinPharmR.pdf#page=31, https://pubmed.ncbi.nlm.nih.gov/18971318/ Page: 31.0	minor
UGT1A8 323	substrate 4014 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2011/021825Orig1s000ClinPharmR.pdf#page=31, https://pubmed.ncbi.nlm.nih.gov/18971318/ Page: 31.0	minor
UGT1A9 423	substrate 4014 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2011/021825Orig1s000ClinPharmR.pdf#page=31, https://pubmed.ncbi.nlm.nih.gov/18971318/ Page: 31.0	minor
UGT1A1 479	substrate 4014 Sources: https://pubmed.ncbi.nlm.nih.gov/18971318/	no
UGT1A3 470	substrate 4014 Sources: https://pubmed.ncbi.nlm.nih.gov/18971318/	no
UGT1A4 399	substrate 4014 Sources: https://pubmed.ncbi.nlm.nih.gov/18971318/	no
UGT1A5 56	substrate 4014 Sources: https://pubmed.ncbi.nlm.nih.gov/18971318/	no
UGT2B10 131	substrate 4014 Sources: https://pubmed.ncbi.nlm.nih.gov/18971318/	no
UGT2B17 237	substrate 4014 Sources: https://pubmed.ncbi.nlm.nih.gov/18971318/	no
UGT2B28 65	substrate 4014 Sources: https://pubmed.ncbi.nlm.nih.gov/18971318/	no
UGT2B4 278	substrate 4014 Sources: https://pubmed.ncbi.nlm.nih.gov/18971318/	no
UGT1A7 249	substrate 4014 Sources: https://pubmed.ncbi.nlm.nih.gov/18971318/	yes
UGT2B15 236	substrate 4014 Sources: https://pubmed.ncbi.nlm.nih.gov/18971318/	yes
UGT2B7 456	substrate 4014 Sources: https://pubmed.ncbi.nlm.nih.gov/18971318/	yes

Tox targets

Target	Modality	Activity	Metabolite	Clinical evidence
hERG 2263	inhibitor 2237 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2011/021825Orig1s000ClinPharmR.pdf#page=73 Page: 73.0

Sourcing

Vendor/Aggregator	ID	URL
ChEBI	CHEBI:68554	https://www.ebi.ac.uk/chebi/searchId.do?chebiId=CHEBI:68554
ChemicalBook	CB0308124	https://www.chemicalbook.com/ChemicalProductProperty_EN_CB0308124
eMolecules	502935	https://www.emolecules.com/cgi-bin/more?vid=502935
MolPort	MolPort-001-792-505	https://www.molport.com/shop/molecule-link/MolPort-001-792-505
Selleck Chemicals	deferiprone	http://www.selleckchem.com/products/deferiprone.html
ZINC	ZINC000000006226	http://zinc15.docking.org/substances/ZINC000000006226

PubMed

Title	Date	PubMed
Combined therapy with deferiprone and desferrioxamine successfully regresses severe heart failure in patients with beta-thalassemia major.	2004-07	14658011
Superoxide, H2O2, and iron are required for TNF-alpha-induced MCP-1 gene expression in endothelial cells: role of Rac1 and NADPH oxidase.	2004-03	14576080
Bone mass and metabolism in thalassemic children and adolescents treated with different iron-chelating drugs.	2004	14691688
Synthesis and antiviral evaluation of 3-hydroxy-2-methylpyridin-4-one dideoxynucleoside derivatives.	2003-12-15	14643328
Polyamine-iron chelator conjugate.	2003-12-04	14640556
Combined therapy with desferrioxamine and deferiprone in thalassemic patients: effect on urinary iron excretion.	2003-12	14687998
Labile plasma iron in iron overload: redox activity and susceptibility to chelation.	2003-10-01	12805056
Pharmacotherapy of iron overload in thalassaemic patients.	2003-10	14521486
Safety and effectiveness of long-term therapy with the oral iron chelator deferiprone.	2003-09-01	12763939
Chromosomal aberration frequencies in patients with thalassaemia major undergoing therapy with deferiprone and deferoxamine in a comparative crossover study.	2003-09	12960415
Do we need more iron-chelating drugs?	2003-08-09	12927446
Pulmonary dysfunction in transfusion-dependent patients with thalassemia major.	2003-07-15	12738605
Role of deferiprone in chelation therapy for transfusional iron overload.	2003-07-01	12637334
Clinical trial of deferiprone iron chelation therapy in beta-thalassaemia/haemoglobin E patients in Thailand.	2003-07	12846901
Iron withdrawal strategies fail to prevent the growth of SiHa-induced tumors in mice.	2003-07	12821347
Deferiprone and hepatic fibrosis.	2003-06-15	12788794
Examination of novel zinc-binding groups for use in matrix metalloproteinase inhibitors.	2003-06-02	12767177
Iron-dependent activation of NF-kappaB in Kupffer cells: a priming mechanism for alcoholic liver disease.	2003-06	12957294
Monitoring long-term efficacy of iron chelation therapy by deferiprone and desferrioxamine in patients with beta-thalassaemia major: application of SQUID biomagnetic liver susceptometry.	2003-06	12786807
Safety monitoring of cardiac and hepatic systems in beta-thalassemia patients with chelating treatment in Taiwan.	2003-06	12756022
Emerging understanding of the advantage of small molecules such as hydroxypyridinones in the treatment of iron overload.	2003-06	12678676
Iron chelator research: past, present, and future.	2003-06	12678671
Signaling role of intracellular iron in NF-kappaB activation.	2003-05-16	12637578
Glutathione depletion increases nitric oxide-induced oxidative stress in primary rat hepatocyte cultures: involvement of low-molecular-weight iron.	2003-05-15	12726916
Potential myocardial iron content evaluation by magnetic resonance imaging in thalassemia major patients treated with Deferoxamine or Deferiprone during a randomized multicenter prospective clinical study.	2003-05	12779268
Comparative effects of deferiprone and deferoxamine on survival and cardiac disease in patients with thalassemia major: a retrospective analysis.	2003-05	12745268
Treatment of cardiac iron overload in thalassemia major.	2003-05	12745264
Multidentate pyridinones inhibit the metabolism of nontransferrin-bound iron by hepatocytes and hepatoma cells.	2003-04	12694182
Comparison between desferrioxamine and combined therapy with desferrioxamine and deferiprone in iron overloaded thalassaemia patients.	2003-04	12670352
The Olivieri case.	2003-02-27	12608401
The Olivieri case.	2003-02-27	12606746
Deferiprone versus desferrioxamine in thalassaemia, and T2* validation and utility.	2003-01-11	12531621
Deferiprone versus desferrioxamine in thalassaemia, and T2* validation and utility.	2003-01-11	12531619
Deferiprone versus desferrioxamine in thalassaemia, and T2* validation and utility.	2003-01-11	12531618
Deferiprone versus desferrioxamine in thalassaemia, and T2* validation and utility.	2003-01-11	12531617
The Olivieri Report--a compelling study of the growing tensions in clinical research.	2003-01	12645235
Patients' health or company profits? The commercialisation of academic research.	2003-01	12645227
Immune status of thalassemic patients receiving deferiprone or combined deferiprone and desferrioxamine chelation treatment.	2003	14663175
Iron deficiency and overload.	2003	14633776
Iron chelation in chemotherapy.	2003	12964245
Benefits and risks of deferiprone in iron overload in Thalassaemia and other conditions: comparison of epidemiological and therapeutic aspects with deferoxamine.	2003	12825969
Clinical research: a tale of two studies.	2003	12813922
Iron chelating agents for treating malaria.	2003	12804409
[Effect of desferrioxamine and deferiprone on osteocalcin secretion in osteoblast-type cells].	2003	12778850
Antihistone and other autoantibodies in beta-thalassemia major patients receiving iron chelators.	2003	12486321
Anti-leukemia activity of 7-hydroxy-2-substituted-methyl-5H-oxazolo[3,2-a]pyrimidin-5-one derivatives.	2002-12	12683674
Experience with the oral iron chelator deferiprone in transfusion-dependent children.	2002-12	12661341
Deferiprone (L1) as an adjuvant therapy for Plasmodium falciparum malaria.	2002-01	12424933
Antimalarial effect of iron chelators.	2002	12572998
Long term deferiprone chelation therapy.	2002	12572992

Patents

Sample Use Guides

In Vivo Use Guide

25 mg/kg to 33 mg/kg body weight, orally, three times per day, for a total daily dose of 75 mg/kg to 99 mg/kg body weight.

Route of Administration: Oral

In Vitro Use Guide

Proliferating CD4+ T cells from control and RRMS subjects, cultured with or without IL-2, decreased in response to 75 μM deferiprone, although the extent of decreased proliferation of CD4+ T cells from RRMS subjects was less than for control subjects. Proliferating CD8+ T cells from control subjects, cultured with or without IL-2, also decreased in response to 75 μM deferiprone, and this decrease was seen in proliferating CD8+ T cells from RRMS cultured with IL-2.

Substance Class	Chemical Created by `admin` on `Mon Mar 31 18:09:00 GMT 2025` Edited by `admin` on `Mon Mar 31 18:09:00 GMT 2025`
Record UNII	2BTY8KH53L
Record Status	`Validated (UNII)`
Record Version

Download

Name

Type

Language

DEFERIPRONE

Official Name

English

APO-066

Preferred Name

English

4(1H)-PYRIDINONE, 3-HYDROXY-1,2-DIMETHYL-

Systematic Name

English

DEFERIPRONE [EP MONOGRAPH]

Common Name

English

NSC-758880

Code

English

L-1

Code

English

3-Hydroxy-1,2-dimethylpyridin-4(1H)-one

Systematic Name

English

APO-66

Code

English

DEFERIPRONE [MART.]

Common Name

English

Deferiprone [WHO-DD]

Common Name

English

L1

Code

English

DEFERIPRONE [ORANGE BOOK]

Common Name

English

3-HYDROXY-1,2-DIMETHYL-4(1H)-PYRIDONE

Systematic Name

English

deferiprone [INN]

Common Name

English

DEFERIPRONE [EMA EPAR]

Common Name

English

CP-20

Code

English

DN-180-01-AF

Code

English

DEFERIPRONE [VANDF]

Common Name

English

DN-18001AF

Code

English

CP20

Code

English

DEFERIPRONE [USAN]

Common Name

English

DEFERIPRONE [MI]

Common Name

English

DEFERIPRON

Common Name

English

PL1

Code

English

PL-1

Code

English

Classification Tree Code System Code

EU-Orphan Drug

EU/3/10/832