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Hydroxyurea (Low Dose)
Low-dose hydroxyurea increases fetal hemoglobin (HbF) production and reduces sickling of red blood cells by inhibiting ribonucleotide reductase.
Low-dose hydroxyurea increases fetal hemoglobin (HbF) production and reduces sickling of red blood cells by inhibiting ribonucleotide reductase. Used for Sickle cell disease (reduction of vaso-occlusive crises and acute chest syndrome), Polycythemia vera, Essential thrombocythemia.
At a glance
| Generic name | Hydroxyurea (Low Dose) |
|---|---|
| Also known as | Hydrea |
| Sponsor | Vanderbilt University Medical Center |
| Drug class | Ribonucleotide reductase inhibitor |
| Target | Ribonucleotide reductase |
| Modality | Small molecule |
| Therapeutic area | Hematology/Oncology |
| Phase | Phase 3 |
Mechanism of action
Hydroxyurea is a ribonucleotide reductase inhibitor that promotes the production of fetal hemoglobin (HbF), which does not polymerize like sickle hemoglobin (HbS). By increasing HbF levels and reducing the proportion of HbS, low-dose hydroxyurea decreases hemolysis, vaso-occlusive crises, and organ damage in sickle cell disease. It also has anti-inflammatory and anti-proliferative effects that contribute to its therapeutic benefit.
Approved indications
- Sickle cell disease (reduction of vaso-occlusive crises and acute chest syndrome)
- Polycythemia vera
- Essential thrombocythemia
Common side effects
- Myelosuppression (anemia, leukopenia, thrombocytopenia)
- Macrocytosis
- Nausea and vomiting
- Headache
- Elevated uric acid/hyperuricemia
Key clinical trials
- Stroke Prevention in Nigeria 2 Trial (PHASE4)
- A Phase 3 Study of Pacritinib in Patients With Primary Myelofibrosis, Post Polycythemia Vera Myelofibrosis, or Post-Essential Thrombocythemia Myelofibrosis (PHASE3)
- Study of Biomarker-Based Treatment of Acute Myeloid Leukemia (PHASE1, PHASE2)
- Galinpepimut-S Versus Investigator's Choice of Best Available Therapy for Maintenance in AML CR2/CRp2 (PHASE3)
- Stroke Prevention in Young Adults With Sickle Cell Anemia
- Reduced Intensity Transplantation for Severe Sickle Cell Disease (PHASE2)
- Personalized, Adaptive Treatment for Locally Advanced Head and Neck Cancer (PHASE1)
- An Efficacy and Safety Study of AG-221 (CC-90007) Versus Conventional Care Regimens in Older Subjects With Late Stage Acute Myeloid Leukemia Harboring an Isocitrate Dehydrogenase 2 Mutation (PHASE3)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Hydroxyurea (Low Dose) CI brief — competitive landscape report
- Hydroxyurea (Low Dose) updates RSS · CI watch RSS
- Vanderbilt University Medical Center portfolio CI