hydroxyurea
Trade name: hydrea
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
Earliest trial started Feb 7, 1984 (NCT00001197)
Timeline
1960s
- Dec 7, 1967
Squibb — Earliest FDA Approval
- Dec 7, 1967
Bristol-Myers Squibb — NDA Secondary Org
- Dec 7, 1967
Squibb — NDA Organization
- Dec 7, 1967
CheplaPharm — Marketing Organization
- Dec 7, 1967
CheplaPharm — NDA Secondary Org
1980s
- Jan 1, 1984
Earliest Phase 2 Sponsor(trial)
1990s
- Jan 1, 1992
Earliest Phase 3 Sponsor(trial)
- Jan 1, 1995
Earliest Phase 1 Sponsor(trial)
- Aug 18, 1995
Roxane Laboratories — Marketing Organization
- Aug 18, 1995
Boehringer Ingelheim — Marketing Organization
- Jul 30, 1998
Barr Laboratories — Marketing Organization
- Feb 24, 1999
Par Pharmaceuticals — Marketing Organization
2010s
- Dec 21, 2017
Addmedica — NDA Secondary Org
2020s
- Apr 8, 2020
Leading Pharma Llc — Marketing Organization
Indications
Approved for
Studied for
- Adrenoleukodystrophy · Phase 2
- Allergy and Immunology · Phase 1/Phase 2
- Anemia · Phase 2
- Anemia, Diamond-Blackfan · Phase 1/Phase 2
- Anemia, Sickle Cell · Phase 4
- Arterial Occlusive Diseases · Phase 2/Phase 3
- Arthritis, Juvenile · Phase 2
- Arthritis, Rheumatoid · Phase 2
- Astrocytoma · Phase 3
- Basal Cell Carcinoma · Phase 1
- beta-Thalassemia · Phase 4
- Bile Duct Neoplasms · Phase 2
- Blood Transfusion · Phase 4
- Bone Marrow Failure Disorders · Phase 1/Phase 2
- Brain Neoplasms · Phase 2
- Carcinoma · Phase 1
- Carcinoma, Adenoid Cystic · Phase 1
- Carcinoma, Mucoepidermoid · Phase 1
- Carcinoma, Squamous Cell · Phase 2/Phase 3
- Carcinoma, Verrucous · Phase 1
- Child · Phase 2
- Congenital Bone Marrow Failure Syndromes · Phase 2
- Drugs, Investigational · Phase 4
- Esophageal Neoplasms · Phase 2
- Fetal Hemoglobin · Phase 2
- Fibroma · Phase 2
- Gangliosidosis, GM1 · Phase 2
- Glioblastoma · Phase 3
- Glioma · Phase 1
- Gliosarcoma · Phase 2
- Graft vs Host Disease · Phase 1
- Granuloma · Phase 1
- Head and Neck Neoplasms · Phase 2
- Healthy Volunteers · Phase 1
- Hematologic Diseases · Phase 3
- Hematopoietic Stem Cell Transplantation · Phase 2/Phase 3
- Hemochromatosis · Phase 3
- Hemoglobinopathies · Phase 3
- Hemoglobin SC Disease · Phase 2
- HIV Infections · Phase 4
- Hodgkin Disease · Phase 2
- Hypertension, Pulmonary · Phase 1/Phase 2
- Idiopathic Pulmonary Fibrosis · Phase 1/Phase 2
- Immune System Diseases · Phase 1/Phase 2
- Inflammation · Phase 2
- Intestinal Neoplasms · Phase 2
- Iron Overload · Phase 2/Phase 3
- Kidney Diseases · Phase 4
- Laryngeal Neoplasms · Phase 3
- Leukemia · Phase 3
- Leukemia, Myelogenous, Chronic, BCR-ABL Positive · Phase 3
- Leukemia, Myeloid, Acute · Phase 2/Phase 3
- Leukemia, Myeloid, Chronic-Phase · Phase 2
- Leukemia, Myelomonocytic, Chronic · Phase 2
- Leukemia, Promyelocytic, Acute · Phase 3
- Leukodystrophy, Globoid Cell · Phase 2
- Leukodystrophy, Metachromatic · Phase 2
- Liver Neoplasms · Phase 2
- Lung Neoplasms · Phase 1
- Lymphocytosis · Phase 2
- Lymphoma · Phase 2
- Lymphoproliferative Disorders · Phase 2
- Malaria · Phase 3
- Meningioma · Phase 2
- Metabolic Diseases · Phase 2
- Mouth Neoplasms · Phase 3
- Mucolipidoses · Phase 2
- Mucopolysaccharidosis III · Phase 2
- Multiple Sclerosis · Phase 2/Phase 3
- Muscular Atrophy, Spinal · Phase 2/Phase 3
- Myelodysplastic Syndromes · Phase 3
- Myeloproliferative Disorders · Phase 3
- Nasopharyngeal Carcinoma · Phase 3
- Nasopharyngeal Neoplasms · Phase 1
- Neoplasm Metastasis · Phase 1
- Neuroblastoma · Phase 1
- Nose Neoplasms · Phase 2
- Oropharyngeal Neoplasms · Phase 1/Phase 2
- Oxidative Stress · Phase 2/Phase 3
- Pancreatic Neoplasms · Phase 2
- Papilloma, Inverted · Phase 1
- Papillomaviridae · Phase 2
- Papillomavirus Infections · Phase 2
- Paranasal Sinus Neoplasms · Phase 3
- Pharyngeal Neoplasms · Phase 3
- Polycythemia Vera · Phase 4
- Primary Immunodeficiency Diseases · Phase 2
- Primary Myelofibrosis · Phase 3
- Pulmonary Disease, Chronic Obstructive · Phase 1/Phase 2
- Renal Insufficiency, Chronic · Phase 1
- Retinal Diseases · Phase 2
- Risk Factors · Phase 3
- Salivary Gland Neoplasms · Phase 1
- Sandhoff Disease · Phase 2
- Severe Acute Malnutrition · Phase 2
- Squamous Cell Carcinoma of Head and Neck · Phase 2
- Stomach Neoplasms · Phase 2
- Stroke · Phase 4
- Tay-Sachs Disease · Phase 2
- Thalassemia · Phase 2
- Thrombocythemia, Essential · Phase 4
- Tongue Neoplasms · Phase 1
- Wolman Disease · Phase 2
Mechanism of action
Ribonucleoside-diphosphate reductase RR1 inhibitor
M2 subunit is binding site
Approval history
- approvedPriority reviewDec 7, 1967
Chemistry & pharmacology
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
Sources
- WikipediaHydroxycarbamide ↗
- NCATSX6Q56QN5QC ↗
- ChEMBLCHEMBL467 ↗
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 ID | Phase | Start date | Sponsor(s) |
|---|---|---|---|
| NCT06930352 | Phase 2 | Jan 10, 2026 | Kura Oncology, Inc., Ohio State University |
| NCT07170592 | Phase 1 | Dec 31, 2025 | Bionoxx Inc. |
| NCT07033598 | Phase 2 | Dec 31, 2025 | Swedish Orphan Biovitrum, Theradex, University of South Florida |
| NCT07190833 | Phase 1 | Nov 30, 2025 | Bionoxx Inc. |
| NCT06871410 | Phase 1 | Nov 15, 2025 | State University of New York, Buffalo |
| NCT07177300 | Phase 4 | Dec 19, 2024 | University of Cincinnati |
| NCT06578507 | Phase 2 | Sep 30, 2024 | Addmedica, University of Paris |
| NCT06606886 | Phase 4 | Sep 16, 2024 | Bahria University |
| NCT06526117 | Phase 4 | Aug 5, 2024 | Aminu Kano Teaching Hospital, Murtala Muhammed Specialist Hospital, National Institute of Neurological Disorders and Stroke (NINDS), Vanderbilt University |
| NCT06456346 | Phase 3 | Jul 16, 2024 | Merck |
| NCT06199557 | Phase 1/Phase 2 (Phase 2) | Jan 25, 2024 | Helse Bergen, University of Bergen |
| NCT07292259 | Phase 2 | Jan 1, 2024 | Government of Pakistan, Pakistan Blood and Marrow Transplant (PBMT) Group |
| NCT06005324 | Phase 1 | Dec 31, 2023 | University of Chicago |
| NCT06093672 | Phase 3 | Dec 31, 2023 | Italfarmaco |
| NCT06299670 | Phase 4 | Nov 1, 2023 | Bangladesh Medical University |
| NCT06171217 | Phase 2 | Oct 27, 2023 | National Heart, Lung, and Blood Institute (NHLBI), University of Cincinnati |
| NCT05853458 | Phase 4 | Jul 28, 2023 | Novartis |
| NCT05953584 | Phase 2 | Jun 20, 2023 | Forma Therapeutics, Inc., Novo Nordisk |
| NCT05451940 | Phase 1/Phase 2 (Phase 2) | Jan 31, 2023 | Carnegie Mellon University, The American Society of Hematology, University of Pittsburgh |
| NCT05285917 | Phase 3 | Dec 31, 2022 | Brown University, National Heart, Lung, and Blood Institute (NHLBI), Novartis |
Organizations
Research & Development (193)
Marketing (12)
| Organization | Org type | Relationship | Date |
|---|---|---|---|
| Addmedica | For profit | NDA2 | Dec 21, 2017 |
| Barr Laboratories | For profit | SYN | Oct 16, 1998 |
| Barr Laboratories | For profit | MKTG | Jul 30, 1998 |
| Boehringer Ingelheim | For profit | MKTG | Aug 18, 1995 |
| Bristol-Myers Squibb | For profit | NDA2 | Dec 7, 1967 |
| CheplaPharm | For profit | MKTG | Dec 7, 1967 |
| CheplaPharm | For profit | NDA2 | Dec 7, 1967 |
| Leading Pharma Llc | For profit | MKTG | Apr 8, 2020 |
| Leading Pharma Llc | For profit | SYN | Apr 8, 2020 |
| Par Pharmaceuticals | For profit | MKTG | Feb 24, 1999 |
| Roxane Laboratories | For profit | MKTG | Aug 18, 1995 |
| Squibb | For profit | NDA | Dec 7, 1967 |