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deferiprone

Trade name: ferriprox

Small moleculeapprovedOrphan Drug FDA

Approved

Oct 14, 2011

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%. — NCATS

Clinical trial activity

58 trials · 58 clinical orgs · 6 marketing orgs

Phase 1
12
Phase 2
24
Phase 3
13
Phase 4
10

Earliest trial started Sep 1, 2000 (NCT00733811)

Timeline

2000s

  1. Dec 12, 2001

    Orphan Drug Designation

  2. Jan 1, 2002

    Earliest Phase 3 Sponsor(trial)

  3. Jan 1, 2005

    Earliest Phase 2 Sponsor(trial)

  4. Jan 1, 2006

    Apotex — Earliest Phase 1 Sponsor(trial)

2010s

  1. Oct 14, 2011

    Apotex — Earliest FDA Approval

  2. Oct 14, 2011

    Chiesi — Marketing Organization

  3. Oct 14, 2011

    Apotex — NDA Secondary Org

  4. Oct 14, 2011

    Apotex — NDA Organization

  5. Oct 14, 2011

    Chiesi — NDA Secondary Org

  6. Feb 8, 2019

    Taro Pharmaceuticals — Marketing Organization

  7. Feb 8, 2019

    Sun Pharmaceuticals — Marketing Organization

2020s

  1. Mar 29, 2021

    Hikma — Marketing Organization

  2. Dec 11, 2025

    Senores Pharms — Marketing Organization

Indications

Approved for

Studied for

Mechanism of action

  • Unspecified targetOTHER

    Unknown

Approval history

  • approvedAcceleratedOct 14, 2011

Chemistry & pharmacology

Loading structure…

SMILES

CN1C=CC(=O)C(O)=C1C
Mol. weight
139.1519 g/mol
Lipinski Ro5
Pass
Rule of 3
Yes
Chirality
Achiral Molecule
Inorganic
No
Polymer
No
Delivery
Oral
Availability
Prescription Only

Oral

Yes

Parenteral

No

Topical

No

Black box warning

Sources

Also known as

  • 1,2-dimethyl-3-hydroxy-4-pyridinone
  • 1,2-dimethyl-3-hydroxypyrid-4-one
  • 1,2-dimethyl-3-hydroxypyrid-4-one
  • 1,2-dimethyl-3-hydroxypyrid-4-one
  • 1,2-dimethyl-3-hydroxypyridin-4-one
  • 3-hydroxy-1,2-dimethyl-4(1h)-pyridone
  • 3-hydroxy-1,2-dimethyl-4(1h)-pyridone
  • 3-hydroxy-1,2-dimethyl-4-pyridinone
  • apo-066
  • apo-066
  • apo-066
  • apo-66
  • apo-66
  • cp20
  • cp20
  • cp20
  • crmd-001
  • crmd-001
  • deferione
  • deferipron
  • deferiprona
  • deferiprone
  • deferiprone
  • deferiprone
  • deferiprone
  • deferiprone
  • deferiprone
  • deferiprone
  • deferiprone
  • deferiprone
  • deferiprone
  • deferiprone
  • deferiprone
  • défériprone
  • défériprone
  • deferiproni
  • deferipronum
  • deferypron
  • defriprone
  • dimethylhydroxypyridone
  • dimethylhydroxypyridone
  • dmohpo
  • dn-180-01-af
  • dn-180-01-af
  • ferriprox
  • ferriprox
  • ferriprox
  • ferriprox
  • ferriprox
  • ferriprox
  • hdmpp cpd
  • hdpp
  • l1
  • l1
  • l1 oral chelate
  • pl1
  • pl1
  • pl-1

Clinical trials

NCT IDPhaseStart dateSponsor(s)
NCT07023666Phase 2Oct 7, 2025Inova Health System
NCT05604131Phase 1Nov 24, 2022Indiana University
NCT05111821Phase 2Jan 1, 2022University of Bordeaux
NCT04184453Early Phase 1 (Phase 1)Nov 26, 2019Fujian Medical University
NCT03754725Phase 2Jul 1, 2019University of Iowa
NCT03802916Phase 2Mar 6, 2019Apotex
NCT03293069Phase 2/Phase 3 (Phase 3)Sep 30, 2018Government of France, University of Lille
NCT03591575Phase 4Aug 1, 2018Apotex
NCT03234686Phase 2Jan 19, 2018Neuroscience Trials Australia
NCT02882477Phase 2/Phase 3 (Phase 3)Dec 1, 2016Hebrew University
NCT02878538N/ASep 1, 2016University of Texas, San Antonio
NCT02980458Phase 1Aug 1, 2016SocraMetrics GmbH, SocraTec R&D GmbH
NCT02728843Phase 2Jul 1, 2016Apotex
NCT02477631Phase 2Feb 1, 2016Bar Ilan University, Hebrew University, Tel Aviv University
NCT02655315Phase 2Feb 1, 2016Apotex, European Commission, University of Lille
NCT02635841N/ADec 17, 2015Apotex
NCT02465489Phase 1Jun 1, 2015Algorithme Pharma Inc, Apotex
NCT02456558Phase 1Jun 1, 2015Apotex
NCT02443545Phase 4May 1, 2015Apotex, University of Pennsylvania
NCT02442310Phase 1May 1, 2015Algorithme Pharma Inc, Apotex
Showing 20 of 58 trials
Page 1 / 3

Organizations

Research & Development (58)

OrganizationOrg typeTrialsAs lead sponsorPhasesEarliest year
ApotexFor profit262252002
Ain Shams UniversityAcademic/Hospital5422007
University of LilleAcademic/Hospital5532009
University of PennsylvaniaAcademic/Hospital4232006
European CommissionGovernment3022012
University of California, San FranciscoAcademic/Hospital3022005
University of LondonAcademic/Hospital3222004
Algorithme Pharma IncFor profit2012015
Consorzio per Valutazioni Biologiche e FarmacologicheAcademic/Hospital2222012
CorMedixFor profit2222010
Fondazione I.R.C.C.S. Istituto Neurologico Carlo BestaAcademic/Hospital2012008
Free University of BrusselsAcademic/Hospital2012008
Hebrew UniversityAcademic/Hospital2122016
La Fundaction Para la Investigacion Biomedica, Madrid, SpainAcademic/Hospital2012008
LipomedFor profit2222002
University of CagliariAcademic/Hospital2012001
University of ParisAcademic/Hospital2012008
Aga Khan UniversityAcademic/Hospital1112014
Azienda Ospedaliera V. CervelloAcademic/Hospital1112000
Bar Ilan UniversityAcademic/Hospital1012016
58 organizations
Page 1 / 3

Marketing (10)

OrganizationOrg typeRelationshipDate
ApotexFor profitNDA2Oct 14, 2011
ApotexFor profitNDAOct 14, 2011
ChiesiFor profitMKTGOct 14, 2011
ChiesiFor profitNDA2Oct 14, 2011
HikmaFor profitMKTGMar 29, 2021
HikmaFor profitSYNMar 29, 2021
Senores PharmsFor profitMKTGDec 11, 2025
Sun PharmaceuticalsFor profitMKTGFeb 8, 2019
Taro PharmaceuticalsFor profitSYNFeb 8, 2019
Taro PharmaceuticalsFor profitMKTGFeb 8, 2019