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
Exploring the "iron shuttle" hypothesis in chelation therapy: effects of combined deferoxamine and deferiprone treatment in hypertransfused rats with labeled iron stores and in iron-loaded rat heart cells in culture.	2001 Aug	11477380
Desferrioxamine-chelatable iron, a component of serum non-transferrin-bound iron, used for assessing chelation therapy.	2001 Feb 1	11157499
Olivieri to testify against Apotex in Europe.	2001 Jun	11385480
[Therapeuetic management of patients with thalassemia major].	2001 May	11475036
Determination of a new oral iron chelator, ICL670, and its iron complex in plasma by high-performance liquid chromatography and ultraviolet detection.	2001 May 5	11393706
The Olivieri dispute: no end in sight?	2002 Feb 19	11873931
I beg to differ.	2002 Jul 9	12137065
Orally active iron chelators.	2002 Jun	12127956
Deferiprone versus deferoxamine in patients with thalassemia major: a randomized clinical trial.	2002 Mar-Apr	12064916
Iron toxicity and chelation therapy.	2002 Oct	12416732
Iron chelating agents for treating malaria.	2003	12804409
Synthesis and antiviral evaluation of 3-hydroxy-2-methylpyridin-4-one dideoxynucleoside derivatives.	2003 Dec 15	14643328
The Olivieri case.	2003 Feb 27	12608401
The Olivieri case.	2003 Feb 27	12606746
Patients' health or company profits? The commercialisation of academic research.	2003 Jan	12645227
Deferiprone versus desferrioxamine in thalassaemia, and T2* validation and utility.	2003 Jan 11	12531619
Iron withdrawal strategies fail to prevent the growth of SiHa-induced tumors in mice.	2003 Jul	12821347
Role of deferiprone in chelation therapy for transfusional iron overload.	2003 Jul 1	12637334
Pulmonary dysfunction in transfusion-dependent patients with thalassemia major.	2003 Jul 15	12738605
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 May	12779268
Signaling role of intracellular iron in NF-kappaB activation.	2003 May 16	12637578
Labile plasma iron in iron overload: redox activity and susceptibility to chelation.	2003 Oct 1	12805056
Safety and effectiveness of long-term therapy with the oral iron chelator deferiprone.	2003 Sep 1	12763939
Bone mass and metabolism in thalassemic children and adolescents treated with different iron-chelating drugs.	2004	14691688

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

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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