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Low Dose Hydroxyurea
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).
At a glance
| Generic name | Low Dose Hydroxyurea |
|---|---|
| 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, the drug decreases hemolysis, vaso-occlusive crises, and organ damage in sickle cell disease. At low doses, it achieves therapeutic benefit while minimizing myelosuppression.
Approved indications
- Sickle cell disease (reduction of vaso-occlusive crises and acute chest syndrome)
Common side effects
- Myelosuppression (anemia, leukopenia, thrombocytopenia)
- Macrocytosis
- Elevated uric acid levels
- Gastrointestinal disturbances
- Headache
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:
- Low Dose Hydroxyurea CI brief — competitive landscape report
- Low Dose Hydroxyurea updates RSS · CI watch RSS
- Vanderbilt University Medical Center portfolio CI