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MolPort-001-792-505
Also known as: 30652-11-0, 3-hydroxy-1,2-dimethyl-4(1h)-pyridone, Ferriprox, 3-hydroxy-1,2-dimethylpyridin-4(1h)-one, Cp20, 1,2-dimethyl-3-hydroxy-4-pyridone
Molecular Formula
C7H9NO2
Molecular Weight
139.15  g/mol
InChI Key
TZXKOCQBRNJULO-UHFFFAOYSA-N
FDA UNII
2BTY8KH53L

A pyridone derivative and iron chelator that is used in the treatment of IRON OVERLOAD in patients with THALASSEMIA.
Deferiprone is an Iron Chelator. The mechanism of action of deferiprone is as an Iron Chelating Activity.
1 2D Structure

MolPort-001-792-505

2 Identification
2.1 Computed Descriptors
2.1.1 IUPAC Name
3-hydroxy-1,2-dimethylpyridin-4-one
2.1.2 InChI
InChI=1S/C7H9NO2/c1-5-7(10)6(9)3-4-8(5)2/h3-4,10H,1-2H3
2.1.3 InChI Key
TZXKOCQBRNJULO-UHFFFAOYSA-N
2.1.4 Canonical SMILES
CC1=C(C(=O)C=CN1C)O
2.2 Other Identifiers
2.2.1 UNII
2BTY8KH53L
2.3 Synonyms
2.3.1 MeSH Synonyms

1. 1,2 Dimethyl 3 Hydroxy 4 Pyridinone

2. 1,2 Dimethyl 3 Hydroxypyrid 4 One

3. 1,2 Dimethyl 3 Hydroxypyridin 4 One

4. 1,2-dimethyl-3-hydroxy-4-pyridinone

5. 1,2-dimethyl-3-hydroxypyrid-4-one

6. 1,2-dimethyl-3-hydroxypyridin-4-one

7. 3 Hydroxy 1,2 Dimethyl 4 Pyridinone

8. 3-hydroxy-1,2-dimethyl-4-pyridinone

9. Dmohpo

10. Ferriprox

11. Hdmpp

2.3.2 Depositor-Supplied Synonyms

1. 30652-11-0

2. 3-hydroxy-1,2-dimethyl-4(1h)-pyridone

3. Ferriprox

4. 3-hydroxy-1,2-dimethylpyridin-4(1h)-one

5. Cp20

6. 1,2-dimethyl-3-hydroxy-4-pyridone

7. 1,2-dimethyl-3-hydroxypyrid-4-one

8. Apo-66

9. 1,2-dimethyl-3-hydroxypyridine-4-one

10. 4(1h)-pyridinone, 3-hydroxy-1,2-dimethyl-

11. Apo-066

12. Dn-180-01-af

13. 3-hydroxy-1,2-dimethylpyridin-4-one

14. Deferipron

15. Mfcd00134497

16. Deferiprone (inn)

17. Pl-1

18. L1

19. Chebi:68554

20. 1,2-dimethyl-3-hydroxy-4(1h)-pyridinone

21. Cp-20

22. Dn-18001af

23. Nsc-758880

24. 2bty8kh53l

25. Mls000069481

26. Hk-1

27. Pl1

28. 4(1h)-pyridinone, 1,2-dimethyl-3-hydroxy-

29. Smr000059136

30. Deferiprone [inn]

31. L-1

32. Cp20 (chelating Agent)

33. Ferriprox (tn)

34. Unii-2bty8kh53l

35. Brn 1447108

36. Deferidone

37. Deferiprona

38. Deferum

39. Deferiprone [usan:inn:ban]

40. Ccris 8318

41. 1,2-dimethyl-3-hydroxy-4-pyridinone

42. 3-hydroxy-1,2-dimethyl-4-pyridinone

43. L-1[chelating Agent]

44. Opera_id_366

45. 3-hydroxy-1,2-dimethyl-4(1h)-pyridinone

46. Deferiprone [mi]

47. 4(1h)-pyridone, 3-hydroxy-1,2-dimethyl-

48. Deferiprone (usan/inn)

49. Deferiprone [usan]

50. Deferiprone [vandf]

51. Deferiprone [mart.]

52. Dsstox_cid_20666

53. Dsstox_rid_79529

54. Dsstox_gsid_40666

55. Schembl94474

56. Deferiprone [who-dd]

57. Mls000758227

58. Mls001424029

59. Mls006011689

60. Chembl70927

61. Deferiprone [ema Epar]

62. Crmd-001

63. Gtpl7456

64. Zinc6226

65. Dtxsid6040666

66. Hsdb 8335

67. Tzxkocqbrnjulo-uhfffaoysa-

68. Deferiprone [orange Book]

69. Ex-a972

70. Cp020

71. Deferiprone [ep Monograph]

72. Hms2051c05

73. Hms2232e24

74. Hms3264p17

75. Hms3393c05

76. Hms3873l13

77. Pharmakon1600-01504512

78. Bcp09606

79. Hy-b0568

80. 3-hydroxy-1,2-dimethylpyrid-4-one

81. Tox21_112429

82. Bbl036482

83. Bdbm50525976

84. Cgp-37391

85. Nsc758880

86. S4067

87. Stl559018

88. 1,2-dimetyl-3-hydroxy-4-pyridinone

89. Akos015902286

90. Ccg-100760

91. Cs-5240

92. Db08826

93. Ds-5493

94. Nc00010

95. Nsc 758880

96. 3-hydroxy-1,2-dimethyl-4- Pyridinone

97. 3-hydroxy-1,2-dimethyl-pyridin-4-one

98. Ncgc00090548-01

99. Ncgc00090548-02

100. Ncgc00090548-04

101. Ncgc00090548-05

102. Ac-33026

103. Bp-12415

104. Sy052576

105. Sbi-0207041.p001

106. Cas-30652-11-0

107. Db-047826

108. 1,2-dimethyl-3-hydroxy-4-pyridone, 99+%

109. Ft-0606424

110. H0944

111. D07416

112. 3-hydroxy-1,2-dimethyl-4(1h)-pyridone, 98%

113. Ab00572605_11

114. Ab00572605_12

115. A820470

116. Q749664

117. Sr-01000721891

118. Deferiprone 1,2-dimethyl-3-hydroxy-4-pyridone

119. Sr-01000721891-4

2.4 Create Date
2005-03-25
3 Chemical and Physical Properties
Molecular Weight 139.15 g/mol
Molecular Formula C7H9NO2
XLogP30.5
Hydrogen Bond Donor Count1
Hydrogen Bond Acceptor Count3
Rotatable Bond Count0
Exact Mass139.063328530 g/mol
Monoisotopic Mass139.063328530 g/mol
Topological Polar Surface Area40.5 Ų
Heavy Atom Count10
Formal Charge0
Complexity228
Isotope Atom Count0
Defined Atom Stereocenter Count0
Undefined Atom Stereocenter Count0
Defined Bond Stereocenter Count0
Undefined Bond Stereocenter Count0
Covalently Bonded Unit Count1
4 Drug and Medication Information
4.1 Drug Information
1 of 2  
Drug NameFerriprox
PubMed HealthDeferiprone (By mouth)
Drug ClassesHeavy Metal Chelator
Drug LabelFerriprox (deferiprone) tablets contain 500mg deferiprone (3-hydroxy-1,2-dimethylpyridin-4-one), a synthetic, orally active, iron-chelating agent. Deferiprone has the following structural formula:Deferiprone is a white to pinkish-white crystallin...
Active IngredientDeferiprone
Dosage FormTablet
RouteOral
Strength500mg
Market StatusPrescription
CompanyApopharma

2 of 2  
Drug NameFerriprox
PubMed HealthDeferiprone (By mouth)
Drug ClassesHeavy Metal Chelator
Drug LabelFerriprox (deferiprone) tablets contain 500mg deferiprone (3-hydroxy-1,2-dimethylpyridin-4-one), a synthetic, orally active, iron-chelating agent. Deferiprone has the following structural formula:Deferiprone is a white to pinkish-white crystallin...
Active IngredientDeferiprone
Dosage FormTablet
RouteOral
Strength500mg
Market StatusPrescription
CompanyApopharma

4.2 Therapeutic Uses

Iron Chelating Agents

National Library of Medicine's Medical Subject Headings. Deferiprone. Online file (MeSH, 2016). Available from, as of June 24, 2016: https://www.nlm.nih.gov/mesh/2016/mesh_browser/MBrowser.html


/CLINICAL TRIALS/ ClinicalTrials.gov is a registry and results database of publicly and privately supported clinical studies of human participants conducted around the world. The Web site is maintained by the National Library of Medicine (NLM) and the National Institutes of Health (NIH). Each ClinicalTrials.gov record presents summary information about a study protocol and includes the following: Disease or condition; Intervention (for example, the medical product, behavior, or procedure being studied); Title, description, and design of the study; Requirements for participation (eligibility criteria); Locations where the study is being conducted; Contact information for the study locations; and Links to relevant information on other health Web sites, such as NLM's MedlinePlus for patient health information and PubMed for citations and abstracts for scholarly articles in the field of medicine. Deferiprone is included in the database.

NIH/NLM; ClinicalTrials.Gov. Available from, as of July 6, 2016: https://clinicaltrials.gov/search/intervention=DEFERIPRONE


Ferriprox (deferiprone) is indicated for the treatment of patients with transfusional iron overload due to thalassemia syndromes when current chelation therapy is inadequate. /Included in US product label/

NIH; DailyMed. Current Medication Information for Ferriprox (Deferiprone) Tablet, Film-coated (Updated: February 2015). Available from, as of July 14, 2016: https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=dc8cbc3d-026c-0db5-42e8-8e93d374dd23


/EXPL THER/ Friedreich ataxia is an inherited disorder characterized by degeneration of the peripheral and central nervous system and hypertrophic cardiomyopathy. Homozygous mutations in the frataxine (FXN) gene reduce expression of frataxin and cause accumulation of iron in the mitochondria. Deferiprone, an oral iron chelator, has been shown effective in cell and animal models of Friedreich ataxia. The results of a 6-month randomized, double blind placebo-controlled study suggested that deferiprone 20 mg/kg/day may reduce disease progression. The authors present their experience of 5 Friedreich ataxia patients treated with deferiprone (20 mg/kg/day), in addition to idebenone treatment, followed over a period of 10-24 months, under off-label authorization. The patients were monitored for laboratory parameters, cardiac assessment, neurological evaluations, and quality of life. The authors conclude that combined therapy of a low dose of deferiprone with idebenone is relatively safe, might improve neurological function, and seems to improve heart hypertrophy, warranting further studies.

PMID:27029487 Elincx-Benizri S et al; J Child Neurol 31 (8): 1036-40 (2016)


/EXPL THER/ Growing body of evidence suggests that Parkinson's disease (PD) is associated with oxidative damage via iron accumulation in the substantia nigra (SN). Low ceruloplasmin (CP)-ferroxidase activity has been identified in the SN and the cerebrospinal fluid (CSF) of patients with PD. The iron chelator, deferiprone, reduces the abnormally high levels of iron in the SN. In order to determine CP's involvement in iron accumulation in SN and PD progression, we aim to compare the ability of iron chelation treatment to reducing both SN iron levels and motor handicap in PD patients according to the level of ceruloplasmin activity. We used a moderate chelation protocol with deferiprone (DFP) based on a, 6-month delayed-start paradigm, randomized placebo controlled clinical trial in 40 PD patients. CP-ferroxidase activity was determined in blood and CSF together with the D544E gene polymorphism (rs701753). Iron levels were determined by R2* MRI sequence and the motor handicap by the UPDRS motor score. After 6 to 12 months of DFP treatment, greater reductions in SN iron levels and UPDRS motor scores were obtained in patients with higher serum and CSF levels of CP-ferroxidase activity. After 6 months of DFP treatment, the AT genotype group displayed greater reduction of iron level in the SN with greater CSF and serum levels of CP activity than the AA genotype group. Although most of the DFP-treated patients displayed clinical and radiological improvements, those with the lower CP activity appeared to respond better to iron chelation. Larger RCTs are now needed to establish whether pharmacological modulation of CP activity could be an innovative neuroprotective strategy in PD.

PMID:25943368 Full text: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4429376 Grolez G et al; BMC Neurol 15:74 (2015)


4.3 Drug Warning

/BOXED WARNING/ WARNING: AGRANULOCYTOSIS/NEUTROPENIA: Ferriprox can cause agranulocytosis that can lead to serious infections and death. Neutropenia may precede the development of agranulocytosis. Measure the absolute neutrophil count (ANC) before starting Ferriprox therapy and monitor the ANC weekly on therapy. Interrupt Ferriprox therapy if neutropenia develops. Interrupt Ferriprox if infection develops, and monitor the ANC more frequently. Advise patients taking Ferriprox to report immediately any symptoms indicative of infection.

NIH; DailyMed. Current Medication Information for Ferriprox (Deferiprone) Tablet, Film-coated (Updated: February 2015). Available from, as of July 14, 2016: https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=dc8cbc3d-026c-0db5-42e8-8e93d374dd23


Based on evidence of genotoxicity and developmental toxicity in animal studies, Ferriprox can cause fetal harm when administered to a pregnant woman. ... If Ferriprox is used during pregnancy or if the patient becomes pregnant while taking Ferriprox, the patient should be apprised of the potential hazard to the fetus.

NIH; DailyMed. Current Medication Information for Ferriprox (Deferiprone) Tablet, Film-coated (Updated: February 2015). Available from, as of July 14, 2016: https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=dc8cbc3d-026c-0db5-42e8-8e93d374dd23


It is not known whether deferiprone is excreted in human milk. Because many drugs are excreted in human milk and because of the potential for adverse reactions in nursing infants from Ferriprox, a decision should be made whether to discontinue nursing or to discontinue the drug, taking into account the importance of the drug to the mother.

NIH; DailyMed. Current Medication Information for Ferriprox (Deferiprone) Tablet, Film-coated (Updated: February 2015). Available from, as of July 14, 2016: https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=dc8cbc3d-026c-0db5-42e8-8e93d374dd23


Safety and effectiveness in elderly individuals have not been established. In general, dose selection for an elderly patient should be cautious, usually starting at the low end of the dosing range, reflecting the greater frequency of decreased hepatic, renal, or cardiac function, and of concomitant disease or other drug therapy.

NIH; DailyMed. Current Medication Information for Ferriprox (Deferiprone) Tablet, Film-coated (Updated: February 2015). Available from, as of July 14, 2016: https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=dc8cbc3d-026c-0db5-42e8-8e93d374dd23


For more Drug Warnings (Complete) data for Deferiprone (13 total), please visit the HSDB record page.


4.4 Drug Indication

Deferiprone is indicated in thalassemia syndromes when first line chelation agents are not adequate to treat transfusional iron overload.


FDA Label


Ferriprox monotherapy is indicated for the treatment of iron overload in patients with thalassaemia major when current chelation therapy is contraindicated or inadequate.

Ferriprox in combination with another chelator is indicated in patients with thalassaemia major when monotherapy with any iron chelator is ineffective, or when prevention or treatment of life-threatening consequences of iron overload (mainly cardiac overload) justifies rapid or intensive correction.


Deferiprone Lipomed monotherapy is indicated for the treatment of iron overload in patients with thalassaemia major when current chelation therapy is contraindicated or inadequate.

Deferiprone Lipomed in combination with another chelator is indicated in patients with thalassaemia major when monotherapy with any iron chelator is ineffective, or when prevention or treatment of life-threatening consequences of iron overload justifies rapid or intensive correction.


Treatment of chronic iron overload


5 Pharmacology and Biochemistry
5.1 MeSH Pharmacological Classification

Iron Chelating Agents

Organic chemicals that form two or more coordination links with an iron ion. Once coordination has occurred, the complex formed is called a chelate. The iron-binding porphyrin group of hemoglobin is an example of a metal chelate found in biological systems. (See all compounds classified as Iron Chelating Agents.)


5.2 FDA Pharmacological Classification
5.2.1 Active Moiety
DEFERIPRONE
5.2.2 FDA UNII
2BTY8KH53L
5.2.3 Pharmacological Classes
Iron Chelator [EPC]; Iron Chelating Activity [MoA]
5.3 ATC Code

V03AC02


V03AC02


V03AC02

S76 | LUXPHARMA | Pharmaceuticals Marketed in Luxembourg | Pharmaceuticals marketed in Luxembourg, as published by d'Gesondheetskeess (CNS, la caisse nationale de sante, www.cns.lu), mapped by name to structures using CompTox by R. Singh et al. (in prep.). List downloaded from https://cns.public.lu/en/legislations/textes-coordonnes/liste-med-comm.html. Dataset DOI:10.5281/zenodo.4587355


V - Various

V03 - All other therapeutic products

V03A - All other therapeutic products

V03AC - Iron chelating agents

V03AC02 - Deferiprone


5.4 Absorption, Distribution and Excretion

Absorption

Deferiprone is absorbed in the upper gastrointestinal tract. Absorption is rapid with maximum plasma concentrations occurring after 1 hour in the fasted state and after 2 hours in the fed state.


Route of Elimination

Within 5-6 hours of administration, more than 90% of deferiprone is eliminated from the plasma. 75 to 90% of deferiprone is excreted in the urine as the metabolite.


Volume of Distribution

In healthy patients, the volume of distribution is 1L/kg, and in thalassemia patients, the volume of distribution is 1.6L/kg.


In healthy subjects, the mean maximum concentration (Cmax) of deferiprone in serum was 20 ug/mL, and the mean total area under the concentration-time curve (AUC) was 53 ug*hr/mL following oral administration of a 1,500 mg dose of Ferriprox tablets in the fasting state. Dose proportionality over the labeled dosage range of 25 to 33 mg/kg three times per day (75 to 99 mg/kg per day) has not been studied. The elimination half life of deferiprone was 1.9 hours. The accumulation of deferiprone and its glucuronide metabolite at the highest approved dosage level of 33 mg/kg three times per day has not been studied. The volume of distribution of deferiprone is 1.6 L/kg in thalassemia patients, and approximately 1 L/kg in healthy subjects. The plasma protein binding of deferiprone in humans is less than 10%.

NIH; DailyMed. Current Medication Information for Ferriprox (Deferiprone) Tablet, Film-coated (Updated: February 2015). Available from, as of July 14, 2016: https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=dc8cbc3d-026c-0db5-42e8-8e93d374dd23


Deferiprone is rapidly absorbed from the upper part of the gastrointestinal tract, appearing in the blood within 5 to 10 minutes of oral administration. Peak serum concentrations occur approximately 1 hour after a single dose in fasted healthy subjects and patients, and up to 2 hours after a single dose in the fed state. Administration with food decreased the Cmax of deferiprone by 38% and the AUC by 10%. While a food effect cannot be ruled out, the magnitude of the exposure change does not warrant dose adjustment.

NIH; DailyMed. Current Medication Information for Ferriprox (Deferiprone) Tablet, Film-coated (Updated: February 2015). Available from, as of July 14, 2016: https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=dc8cbc3d-026c-0db5-42e8-8e93d374dd23


More than 90% of deferiprone is eliminated from plasma within 5 to 6 hours of ingestion. Following oral administration, 75% to 90% is recovered in the urine in the first 24 hours, primarily as metabolite.

NIH; DailyMed. Current Medication Information for Ferriprox (Deferiprone) Tablet, Film-coated (Updated: February 2015). Available from, as of July 14, 2016: https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=dc8cbc3d-026c-0db5-42e8-8e93d374dd23


/MILK/ It is not known whether deferiprone is excreted in human milk.

NIH; DailyMed. Current Medication Information for Ferriprox (Deferiprone) Tablet, Film-coated (Updated: February 2015). Available from, as of July 14, 2016: https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=dc8cbc3d-026c-0db5-42e8-8e93d374dd23


For more Absorption, Distribution and Excretion (Complete) data for Deferiprone (8 total), please visit the HSDB record page.


5.5 Metabolism/Metabolites

Deferiprone is mainly metabolized by UGT1A6 to the 3-O-glucuronide metabolite. This metabolite cannot chelate iron.


In humans, the majority of the deferiprone is metabolized, primarily by UGT1A6. The contribution of extrahepatic (e.g., renal) UGT1A6 is unknown. The major metabolite of deferiprone is the 3-O-glucuronide, which lacks iron binding capability. Peak serum concentration of the glucuronide occurs 2 to 4 hours after administration of deferiprone in fasting subjects.

NIH; DailyMed. Current Medication Information for Ferriprox (Deferiprone) Tablet, Film-coated (Updated: February 2015). Available from, as of July 14, 2016: https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=dc8cbc3d-026c-0db5-42e8-8e93d374dd23


5.6 Biological Half-Life

The half-life is 1.9 hours.


The pharmacokinetics of deferiprone in children was assessed in 7 patients with thalassemia and iron overload aged 11 to 18 years (mean age= 15 + or - 2.7 years; median=16 years). These patients were on long term therapy with deferiprone and were thus considered to be at steady state. ... Serum levels of deferiprone were maximal approximately 2 hours after dosing and declined with a half-life of 1.8 hours; levels of deferiprone glucuronide peaked at approximately 3 hours and fell with a half-life of 2.0 hours. ...

Health Canada; Product Monograph for Ferriprox (Deferiprone) Tablets (500 mg and 1000 mg) Deferiprone Oral Solution (100 mg/mL). Drug Identification Number (DIN): 02436531 p.17 (Date of Preparation: January 22, 2015). Available from, as of July 14, 2016: https://webprod5.hc-sc.gc.ca/dpd-bdpp/start-debuter.do?lang=eng


In healthy subjects ... following oral administration of a 1,500 mg dose of Ferriprox tablets in the fasting state ... the elimination half life ... was 1.9 hours.

NIH; DailyMed. Current Medication Information for Ferriprox (Deferiprone) Tablet, Film-coated (Updated: February 2015). Available from, as of July 14, 2016: https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=dc8cbc3d-026c-0db5-42e8-8e93d374dd23


5.7 Mechanism of Action

Deferiprone is an iron chelator that binds to ferric ions (iron III) and forms a 3:1 (deferiprone:iron) stable complex and is then eliminated in the urine. Deferiprone is more selective for iron in which other metals such as zinc, copper, and aluminum have a lower affinity for deferiprone.


Deferiprone is a chelating agent with an affinity for ferric ion (iron III). Deferiprone binds with ferric ions to form neutral 3:1 (deferiprone:iron) complexes that are stable over a wide range of pH values. Deferiprone has a lower binding affinity for other metals such as copper, aluminum and zinc than for iron.

NIH; DailyMed. Current Medication Information for Ferriprox (Deferiprone) Tablet, Film-coated (Updated: February 2015). Available from, as of July 14, 2016: https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=dc8cbc3d-026c-0db5-42e8-8e93d374dd23


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