hydroxyurea

Trade name: hydrea

Small moleculeapproved

Approved

Dec 7, 1967

Hydroxyurea is an oral antimetabolite; inhibits ribonucleotide reductase and DNA synthesis. It is used for resistant chronic myeloid leukemia, locally advanced squamous cell carcinomas of the head and neck (excluding lip) in combination with concurrent chemoradiation, and to reduce the frequency of painful crises and the need for blood transfusions in patients with sickle cell anemia with recurrent moderate to severe painful crises. Hydroxyurea, a myelosuppressive agent, is the only effective drug proven to reduce the frequency of painful episodes. It raises the level of HbF and the haemoglobin level. It usually decreases the rate of painful episodes by 50%. It was first tested in sickle cell disease in 1984. It also decreases the rate of ACS episodes and blood transfusions by ~50 % in adults. It was developed as an anticancer drug and has been used to treat myeloproliferative syndromes-leukemia, melanoma, and ovarian cancer. It was approved for use by FDA in adults. Side effects includes neutropenia, bone marrow suppression, elevation of hepatic enzymes, anorexia, nausea, vomiting and infertility. — NCATS

Clinical trial activity

193 trials · 193 clinical orgs · 9 marketing orgs

Phase 1
43
Phase 2
130
Phase 3
47
Phase 4
12

Earliest trial started Feb 7, 1984 (NCT00001197)

Timeline

1960s

  1. Dec 7, 1967

    Squibb — Earliest FDA Approval

  2. Dec 7, 1967

    Bristol-Myers Squibb — NDA Secondary Org

  3. Dec 7, 1967

    Squibb — NDA Organization

  4. Dec 7, 1967

    CheplaPharm — Marketing Organization

  5. Dec 7, 1967

    CheplaPharm — NDA Secondary Org

1980s

  1. Jan 1, 1984

    Earliest Phase 2 Sponsor(trial)

1990s

  1. Jan 1, 1992

    Earliest Phase 3 Sponsor(trial)

  2. Jan 1, 1995

    Earliest Phase 1 Sponsor(trial)

  3. Aug 18, 1995

    Roxane Laboratories — Marketing Organization

  4. Aug 18, 1995

    Boehringer Ingelheim — Marketing Organization

  5. Jul 30, 1998

    Barr Laboratories — Marketing Organization

  6. Feb 24, 1999

    Par Pharmaceuticals — Marketing Organization

2010s

  1. Dec 21, 2017

    Addmedica — NDA Secondary Org

2020s

  1. Apr 8, 2020

    Leading Pharma Llc — Marketing Organization

Indications

Studied for

Mechanism of action

Approval history

  • approvedPriority reviewDec 7, 1967

Chemistry & pharmacology

Loading structure…

SMILES

NC(=O)NO
Mol. weight
76.0547 g/mol
Lipinski Ro5
Pass
Rule of 3
No
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

  • biosupressin
  • carbamohydroxamic acid
  • carbamohydroxamic acid
  • carbamohydroxamic acid
  • carbamohydroximic acid
  • carbamohydroximic acid
  • carbamohydroximic acid
  • carbamoyl oxime
  • carbamoyl oxime
  • carbamoyl oxime
  • carbamyl hydroxamate
  • carbamyl hydroxamate
  • droxia
  • droxia
  • hidroxicarbamida
  • hidroxicarbamida
  • hydrea
  • hydrea
  • hydrea
  • hydrea
  • hydroxurea
  • hydroxycarbamid
  • hydroxycarbamid
  • hydroxycarbamid
  • hydroxycarbamide
  • hydroxycarbamide
  • hydroxycarbamide
  • hydroxycarbamide
  • hydroxycarbamide
  • hydroxycarbamide
  • hydroxycarbamide
  • hydroxycarbamidum
  • hydroxycarbamidum
  • hydroxyharnstoff
  • hydroxyharnstoff
  • hydroxyurea
  • hydroxyurea
  • hydroxyurea
  • hydroxyurea
  • hydroxyurea
  • hydroxyurea
  • hydroxyurea
  • hydroxyurea
  • n-carbamoylhydroxylamine
  • n-carbamoylhydroxylamine
  • n-hydroxyurea
  • n-hydroxyurea
  • oxyrea
  • oxyurea
  • oxyurea
  • oxyurea
  • siklos
  • siklos
  • siklos
  • siklos
  • siklos
  • siklos
  • sq-1089
  • sq-1089

Clinical trials

NCT IDPhaseStart dateSponsor(s)
NCT06930352Phase 2Jan 10, 2026Kura Oncology, Inc., Ohio State University
NCT07170592Phase 1Dec 31, 2025Bionoxx Inc.
NCT07033598Phase 2Dec 31, 2025Swedish Orphan Biovitrum, Theradex, University of South Florida
NCT07190833Phase 1Nov 30, 2025Bionoxx Inc.
NCT06871410Phase 1Nov 15, 2025State University of New York, Buffalo
NCT07177300Phase 4Dec 19, 2024University of Cincinnati
NCT06578507Phase 2Sep 30, 2024Addmedica, University of Paris
NCT06606886Phase 4Sep 16, 2024Bahria University
NCT06526117Phase 4Aug 5, 2024Aminu Kano Teaching Hospital, Murtala Muhammed Specialist Hospital, National Institute of Neurological Disorders and Stroke (NINDS), Vanderbilt University
NCT06456346Phase 3Jul 16, 2024Merck
NCT06199557Phase 1/Phase 2 (Phase 2)Jan 25, 2024Helse Bergen, University of Bergen
NCT07292259Phase 2Jan 1, 2024Government of Pakistan, Pakistan Blood and Marrow Transplant (PBMT) Group
NCT06005324Phase 1Dec 31, 2023University of Chicago
NCT06093672Phase 3Dec 31, 2023Italfarmaco
NCT06299670Phase 4Nov 1, 2023Bangladesh Medical University
NCT06171217Phase 2Oct 27, 2023National Heart, Lung, and Blood Institute (NHLBI), University of Cincinnati
NCT05853458Phase 4Jul 28, 2023Novartis
NCT05953584Phase 2Jun 20, 2023Forma Therapeutics, Inc., Novo Nordisk
NCT05451940Phase 1/Phase 2 (Phase 2)Jan 31, 2023Carnegie Mellon University, The American Society of Hematology, University of Pittsburgh
NCT05285917Phase 3Dec 31, 2022Brown University, National Heart, Lung, and Blood Institute (NHLBI), Novartis
Showing 20 of 193 trials
Page 1 / 10

Organizations

Research & Development (193)

OrganizationOrg typeTrialsAs lead sponsorPhasesEarliest year
National Cancer Institute (NCI)Government22731994
National Heart, Lung, and Blood Institute (NHLBI)Government18841984
University of CincinnatiAcademic/Hospital141152011
University of ChicagoAcademic/Hospital121031996
Duke UniversityAcademic/Hospital10742000
St. Jude Children's HospitalAcademic/Hospital10731996
NovartisFor profit9342004
Vanderbilt UniversityAcademic/Hospital8742011
University of PittsburghAcademic/Hospital7532012
AddmedicaFor profit6532015
National Institute of Allergy and Infectious Diseases (NIAID)Government6631999
Northwestern UniversityAcademic/Hospital6421999
Aminu Kano Teaching HospitalAcademic/Hospital5042013
Baylor UniversityAcademic/Hospital5332006
Bristol-Myers SquibbFor profit5421997
National Institutes of Health (NIH)Government5131995
Children's National Medical CenterAcademic/Hospital4222000
Emory UniversityAcademic/Hospital4122000
Harvard UniversityAcademic/Hospital4131997
Johns Hopkins UniversityAcademic/Hospital4121992
193 organizations
Page 1 / 10

Marketing (12)

OrganizationOrg typeRelationshipDate
AddmedicaFor profitNDA2Dec 21, 2017
Barr LaboratoriesFor profitSYNOct 16, 1998
Barr LaboratoriesFor profitMKTGJul 30, 1998
Boehringer IngelheimFor profitMKTGAug 18, 1995
Bristol-Myers SquibbFor profitNDA2Dec 7, 1967
CheplaPharmFor profitMKTGDec 7, 1967
CheplaPharmFor profitNDA2Dec 7, 1967
Leading Pharma LlcFor profitMKTGApr 8, 2020
Leading Pharma LlcFor profitSYNApr 8, 2020
Par PharmaceuticalsFor profitMKTGFeb 24, 1999
Roxane LaboratoriesFor profitMKTGAug 18, 1995
SquibbFor profitNDADec 7, 1967