Drugs / Deferiprone
last change Jun 2026 re-read 2 minutes ago

Deferiprone

Small molecule targets DOHH via inhibition

Developed for
disorder of iron metabolism and transport · beta-thalassemia major · hemosiderosis · pantothenate kinase-associated neurodegeneration · sickle cell disease · acute kidney injury · amyotrophic lateral sclerosis · anemia
+15 more · beta thalassemia · Friedreich ataxia · aceruloplasminemia · acquired aneurysmal subarachnoid hemorrhage · Alzheimer disease · cognitive impairment with or without cerebellar ataxia · heart disorder · HIV infectious disease · iron metabolism disease · myelodysplastic syndrome · neurodegenerative disease · Parkinson disease · stroke disorder · acute myocardial infarction · cardiomyopathy
Investigated by
ApoPharma · Chiesi Canada Corp · Children's Hospital of Philadelphia · Consorzio per Valutazioni Biologiche e Farmacologiche · CorMedix · European Commission
+27 more · Hadassah Medical Organization · Lipomed · The Cooley's Anemia Foundation, · University Hospital, Lille · Algorithme Pharma Inc · Ann & Robert H Lurie Children's Hospital of Chicago · Apotex Inc. · Azienda Ospedaliera V. Cervello · CORDA, The Heart Charity · Carelon Research · China Medical University Hospital · Duke University · Ente Ospedaliero Ospedali Galliera · First Affiliated Hospital of Fujian Medical University · Food and Drug Administration (FDA) · Inova Health Care Services · Kaplan Medical Center · Ministry of Health, France · National Heart, Lung, and Blood Institute (NHLBI) · Neuroscience Trials Australia · Royal Brompton & Harefield NHS Foundation Trust · Sheba Medical Center · Tel Aviv Medical Center · The Florey Institute of Neuroscience and Mental Health · The UK Thalassemia Society · University Hospital, Bordeaux · Ziv Medical Center

Regulatory milestones approvals, filings & regulatory actions · 5 recorded

MilestoneJurisdictionBrandIndicationDateSentence it was read from
Label expansions (3) US (FDA) FERRIPROX 2018-04-20 – 2021-04-30 fda.gov
Approved US (FDA) FERRIPROX 2011-10-14 fda.gov
Approved EU (EMA) Ferriprox 1999-08-25 europa.eu

Trials 43 · a red edge is where a trial was stopped

PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 01 trial
Phase 0 NCT04184453 Dec 2019 → Oct 2022 aceruloplasminemia First Affiliated Hospital of Fujian Medical University Unknown No outcome recorded
Phase 16 trials
Phase 1 NCT02465489 Jun → Aug 2015 ApoPharma Completed No outcome recorded
Phase 1 NCT02456558 Jun 2015 → Mar 2016 ApoPharma Completed No outcome recorded
Phase 1 NCT02442310 May → Jul 2015 ApoPharma Completed No outcome recorded
Phase 1 NCT02189941 May → Jun 2014 ApoPharma Completed No outcome recorded
Phase 1 NCT01989455 Nov 2013 → Mar 2014 ApoPharma Completed No outcome recorded
Phase 1 NCT02191657 Nov 2006 → May 2008 HIV infectious disease ApoPharma Completed No outcome recorded
Phase 1/23 trials
Phase 1/2 NCT03754725 Oct 2020 → Jun 2026 overdue acquired aneurysmal subarachnoid hemorrhage, dementia Duke University Active not recruiting No outcome recorded
Phase 1/2 NCT01709032 Sep 2012 → Aug 2015 beta-thalassemia major Children's Hospital of Philadelphia Completed No outcome recorded
Phase 1/2 NCT00530127 Apr 2008 → Jul 2009 Friedreich ataxia ApoPharma Completed No outcome recorded
Phase 213 trials
Phase 2 NCT07023666 Oct 2025 → Jun 2026 overdue sickle cell disease Inova Health Care Services Recruiting No outcome recorded
Phase 2 NCT05111821 Jun 2022 → Apr 2025 stroke disorder University Hospital, Bordeaux Terminated No outcome recorded
Phase 2 NCT03802916 Mar → Dec 2019 hemosiderosis ApoPharma Completed No outcome recorded
Phase 2 NCT03234686 Jan 2018 → Jan 2023 Alzheimer disease, cognitive impairment with or without cerebellar ataxia Neuroscience Trials Australia Completed No outcome recorded
Phase 2 NCT02728843 Oct 2016 → Aug 2019 Parkinson disease ApoPharma Completed No outcome recorded
Phase 2 NCT02477631 Feb 2016 → May 2017 hemosiderosis, myelodysplastic syndrome Sheba Medical Center Completed No outcome recorded
Phase 2 NCT02164253 Sep 2013 → Dec 2016 amyotrophic lateral sclerosis, disorder of iron metabolism and transport University Hospital, Lille Completed No outcome recorded
Phase 2 NCT01740713 Dec 2012 → Dec 2013 disorder of iron metabolism and transport Consorzio per Valutazioni Biologiche e Farmacologiche Completed No outcome recorded
Phase 2 NCT01146925 Jun 2010 → Jun 2011 acute kidney injury CorMedix Completed No outcome recorded
Phase 2 NCT00897221 Jun 2009 → Mar 2011 Friedreich ataxia ApoPharma Completed No outcome recorded
Phase 2/32 trials
Phase 2/3 NCT03293069 Jan 2019 → Nov 2023 amyotrophic lateral sclerosis University Hospital, Lille Completed No outcome recorded
Phase 2/3 NCT02882477 Dec 2016 → May 2018 diabetes mellitus, iron metabolism disease, optic atrophy, peptic ulcer disease +2 Hadassah Medical Organization Unknown No outcome recorded
Phase 36 trials
Phase 3 NCT02174848 Jun 2014 → Mar 2018 pantothenate kinase-associated neurodegeneration ApoPharma Completed No outcome recorded
Phase 3 NCT01825512 Mar 2014 → Sep 2017 disorder of iron metabolism and transport Consorzio per Valutazioni Biologiche e Farmacologiche Completed No outcome recorded
Phase 3 NCT01741532 Dec 2012 → Oct 2016 pantothenate kinase-associated neurodegeneration ApoPharma Completed No outcome recorded
Phase 3 NCT01391520 Jan 2012 → Mar 2013 acute kidney injury CorMedix Withdrawn No outcome recorded
Phase 3 NCT00529152 Aug 2007 → Jul 2008 disorder of iron metabolism and transport ApoPharma Completed No outcome recorded
Phase 3 NCT00350662 Jan 2002 → ? disorder of iron metabolism and transport Lipomed Completed No outcome recorded
Phase 49 trials
Phase 4 NCT03591575 Nov 2018 → Sep 2020 beta-thalassemia major, disorder of iron metabolism and transport Chiesi Canada Corp Completed No outcome recorded
Phase 4 NCT02443545 May 2015 → Apr 2019 anemia, disorder of iron metabolism and transport, sickle cell disease ApoPharma Terminated No outcome recorded
Phase 4 NCT02041299 Apr 2014 → Apr 2019 anemia, disorder of iron metabolism and transport, sickle cell disease ApoPharma Terminated No outcome recorded
Phase 4 NCT01767103 Jan 2013 → Apr 2014 ApoPharma Completed No outcome recorded
Phase 4 NCT01770652 Jan → Jul 2013 impaired renal function disease ApoPharma Completed No outcome recorded
Phase 4 NCT01860703 Nov → Dec 2012 ApoPharma Completed No outcome recorded
Phase 4 NCT00103753 May 2004 → Jun 2005 beta thalassemia Royal Brompton & Harefield NHS Foundation Trust Unknown No outcome recorded
Phase 4 NCT00105495 Dec 2002 → Oct 2004 beta-thalassemia major, hemosiderosis ApoPharma Completed No outcome recorded
Phase 4 NCT00733811 Sep 2000 → Jan 2008 beta-thalassemia major Azienda Ospedaliera V. Cervello Completed No outcome recorded
Phase not applicable1 trial
NCT02198508 Jul 2007 → Jul 2008 beta-thalassemia major China Medical University Hospital Completed No outcome recorded
Phase not stated2 trials
NCT00293098 Mar 2006 → ? disorder of iron metabolism and transport Children's Hospital of Philadelphia Approved for marketing No outcome recorded
NCT02635841 pantothenate kinase-associated neurodegeneration Chiesi Canada Corp No longer available No outcome recorded
Also used as a comparator or background therapy in 14 trials
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 01 trial
Phase 0 NCT02878538 Comparator Jan 2018 → Apr 2022 cognitive impairment with or without cerebellar ataxia The University of Texas Health Science Center at San Antonio Withdrawn No outcome recorded
Phase 11 trial
Phase 1 NCT01835496 Comparator May 2013 → Apr 2014 sickle cell disease ApoPharma Completed No outcome recorded
Phase 24 trials
Phase 2 NCT07292259 Background Jan 2024 → Dec 2025 beta-thalassemia major Pakistan Blood and Marrow Transplant (PBMT) Group Completed No outcome recorded
Phase 2 NCT05604131 Comparator Nov 2022 → Mar 2029 expected acute myocardial infarction Rohan Dharmakumar Recruiting No outcome recorded
Phase 2 NCT02655315 Comparator Feb 2016 → Sep 2020 Parkinson disease University Hospital, Lille Completed No outcome recorded
Phase 2 NCT01539837 Comparator Feb 2012 → Sep 2014 Parkinson disease Imperial College London Completed No outcome recorded
Phase 2/34 trials
Phase 2/3 NCT02173951 Comparator Jul 2014 → Jul 2015 beta-thalassemia major Ain Shams University Unknown No outcome recorded
Phase 2/3 NCT02065492 Background Feb 2014 → Oct 2015 thalassemia Aga Khan University Completed No outcome recorded
Phase 2/3 NCT01511848 Comparator Feb 2012 → Feb 2013 beta-thalassemia major, hemosiderosis, sickle cell disease Ain Shams University Unknown No outcome recorded
Phase 2/3 NCT00943748 Comparator Oct 2009 → Aug 2011 Parkinson disease University Hospital, Lille Completed No outcome recorded
Phase 41 trial
Phase 4 NCT00800761 Comparator Dec 2001 → Jun 2006 cardiomyopathy, disorder of iron metabolism and transport Ospedale Microcitemico Completed No outcome recorded
Phase not applicable3 trials
NCT01996683 Comparator Nov 2014 → Nov 2015 beta thalassemia Ain Shams University Unknown No outcome recorded
NCT02880033 Comparator Feb 2011 → Oct 2019 Parkinson disease, amyotrophic lateral sclerosis, disorder of iron metabolism and transport University Hospital, Lille Completed No outcome recorded
NCT01597765 Background Jun 2009 → Jun 2011 thalassemia Mahidol University Completed No outcome recorded

Evidence & citations 21 cited values

Every value below carries the sentence it was read from. 63 sources stand behind the page.

FieldValueCited text
Known as deferiprone “Comparison of safety, efficacy, compliance, treatment satisfaction, and quality of life (QoL) of two regimens: deferiprone (DFP) and deferoxamine (DFO) versus DFP and...” PMID 25600572 Mar 2015
53

“This will comprise of standard chelation drugs (Deferasirox or Deferoxamine or Combination of Deferoxamine and Deferiprone).” NCT02065492

“Patients will also continue the iron chelation therapy (Deferasirox, Deferiprone or Deferoxamine) in case of iron overload.” NCT07292259

NCT02880033

NCT01709032

NCT02882477

NCT02191657

NCT01770652

NCT02173951

NCT02456558

NCT02477631

NCT02878538

NCT07023666

NCT04184453

NCT00530127

NCT01741532

NCT01989455

NCT01539837

NCT00115349

NCT02041299

NCT00350662

NCT00105495

NCT03293069

NCT02728843

NCT00349453

NCT02442310

NCT02189941

NCT01146925

NCT01860703

NCT00529152

NCT02174848

NCT02164253

NCT02465489

NCT01825512

NCT03591575

NCT01391520

NCT00943748

NCT00103753

NCT00897221

NCT02443545

NCT02635841

NCT05604131

NCT03234686

NCT02198508

NCT00800761

NCT00293098

NCT02655315

NCT00907283

NCT03754725

NCT01597765

NCT05111821

NCT00733811

NCT03802916

NCT01740713

Known as 1,2 dimethyl-3-hydroxypyrid-4-one “Deferiprone (L1; CP20; 1,2 dimethyl-3-hydroxy-pyrid-4-one) is an orally active iron chelator investigated in various clinical trials since 1987.” NCT00350662
1

“Deferiprone (L1; CP20; 1,2 dimethyl-3-hydroxypyrid-4-one) is an orally active iron chelator investigated in various clinical trials since 1987.” NCT00349453

Known as 1,2-dimethyl-3-hydroxypyridin-4-one “We recently demonstrated (for the first time) the feasibility, efficacy and acceptability of the conservative iron chelation approach in pilot translational studies in...” NCT02655315
Known as APO-066 ChEMBL registry synonym — accepted as the source's own label CHEMBL70927
Known as APO-66 ChEMBL registry synonym — accepted as the source's own label CHEMBL70927
Known as CP20 “Deferiprone (L1; CP20; 1,2 dimethyl-3-hydroxy-pyrid-4-one) is an orally active iron chelator investigated in various clinical trials since 1987.” NCT00350662
1

“Deferiprone (L1; CP20; 1,2 dimethyl-3-hydroxypyrid-4-one) is an orally active iron chelator investigated in various clinical trials since 1987.” NCT00349453

Known as CRMD-001 “This trial will evaluate whether treatment with CRMD-001 (unique formulations of the iron chelator, Deferiprone) will reduce the incidence of AKI in subjects with CKD and...” NCT01146925
1

“This trial will evaluate whether treatment with CRMD001 (unique formulations of the iron chelator, Deferiprone) will reduce morbidity and mortality” NCT01391520

Known as Deferipron ChEMBL registry synonym — accepted as the source's own label CHEMBL70927
Known as Deferiprona ChEMBL registry synonym — accepted as the source's own label CHEMBL70927
Known as deferiprone 50 mg/kg/day brand name GPO-L-ONE deferiprone 50 mg/kg/day brand name GPO-L-ONENCT01597765
Known as Deferiprone lipomed ChEMBL registry synonym — accepted as the source's own label CHEMBL70927
Known as DFP “Comparison of safety, efficacy, compliance, treatment satisfaction, and quality of life (QoL) of two regimens: deferiprone (DFP) and deferoxamine (DFO) versus DFP and...” PMID 25600572 Mar 2015
5

“Half of participants will receive the deferiprone (DFP) to 15 mg / kg twice daily morning and evening (30mg / kg per day).The treatment lasts nine months.” NCT02655315

“we assessed the effect of oxidative insults in mice systemically prechelated with deferiprone (DFP) by following motor functions” PMID 24251381 Feb 2014

“oral deferiprone (DFP) with those of combinations of parenteral desferrioxamine (DFO) with oral DFP.” PMID 18055982 Dec 2007

“starting dose of Deferiprone (DFP) 50mg⁄ kg ⁄ d, administered orally 3 times daily.” NCT02173951

DFP(Deferiprone)” NCT02198508

Known as DFX “Comparison of safety, efficacy, compliance, treatment satisfaction, and quality of life (QoL) of two regimens: deferiprone (DFP) and deferoxamine (DFO) versus DFP and...” PMID 25600572 Mar 2015
Known as DN-180-01-AF ChEMBL registry synonym — accepted as the source's own label CHEMBL70927
Known as Ferriprox “Drug: Ferriprox It will be given orally in the morning Other Name: DFP Initial dose 25/mg/kg in 3 divided doses (better tolerated if started and then built up over 4 weeks).” NCT01511848
7

“Safety and Efficacy of Ferriprox™ (Deferiprone) Oral Solution in Iron Overloaded Pediatric Patients” NCT00529152

“who are currently taking deferiprone immediate-release tablets (Ferriprox) three times a day” NCT03802916

Ferriprox (deferiprone) 500 mg immediate release tablet formulation” NCT02465489

Ferriprox (deferiprone)” NCT00105495

NCT01996683

NCT01835496

NCT01767103

Known as Ferrirpox “long-term chelation through Deferiprone (Ferrirpox®, Chiesi) administered daily from 3-to-5 days following stroke to 6 months” NCT05111821
Known as NSC-758880 ChEMBL registry synonym — accepted as the source's own label CHEMBL70927
Action Inhibit “deferiprone produced infection-independent deficiency of hydroxylated hypusyl-eIF5A.” PMID 27191165 May 2016
Modality Small molecule “Deferiprone presents chemical-physical characteristics (low molecular weight, favourable octanol:water partition coefficient, neutral charge)” NCT00907283
Route Oral “Fifteen of these patients received combination therapy with subcutaneous deferoxamine and oral deferiprone with CMR follow-up.” PMID 18298856 Feb 2008
Target DOHH “The uniform deferiprone threshold is in agreement with mapping of, and crystallographic 3D-data on, the active site of deoxyhypusyl hydroxylase (DOHH), the eIF5A-hydroxylating enzyme.” PMID 27191165 May 2016