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Hydroxyurea (HU)
Hydroxyurea inhibits ribonucleotide reductase, reducing deoxyribonucleotide synthesis and slowing DNA replication in rapidly dividing cells.
Hydroxyurea inhibits ribonucleotide reductase, reducing deoxyribonucleotide synthesis and slowing DNA replication in rapidly dividing cells. Used for Chronic myeloid leukemia (CML), Sickle cell disease (to reduce vaso-occlusive crises), Polycythemia vera.
At a glance
| Generic name | Hydroxyurea (HU) |
|---|---|
| Sponsor | Bionoxx Inc. |
| Drug class | Antimetabolite; ribonucleotide reductase inhibitor |
| Target | Ribonucleotide reductase |
| Modality | Small molecule |
| Therapeutic area | Oncology; Hematology |
| Phase | Phase 3 |
Mechanism of action
By blocking the enzyme ribonucleotide reductase, hydroxyurea depletes the deoxyribonucleotide pool required for DNA synthesis, thereby slowing or halting cell division. In sickle cell disease, it also increases fetal hemoglobin (HbF) production, which inhibits polymerization of sickle hemoglobin and reduces hemolysis. This dual mechanism makes it useful in both oncology and hematologic disorders.
Approved indications
- Chronic myeloid leukemia (CML)
- Sickle cell disease (to reduce vaso-occlusive crises)
- Polycythemia vera
- Essential thrombocythemia
Common side effects
- Myelosuppression (anemia, leukopenia, thrombocytopenia)
- Nausea and vomiting
- Elevated uric acid (tumor lysis)
- Peripheral neuropathy
- Skin hyperpigmentation
Key clinical trials
- Study on Efficacy and Safety of Givinostat Versus Hydroxyurea in Patients With Polycythemia Vera (PHASE3)
- Evaluation of HU-resistance in Adult Patients With Polycythemia Vera Who Meet PV-AIM Predictors (PHASE4)
- Prophylactic Transfusion In Pregnant in Women With Sickle Cell Disease (PHASE4)
- Research Study Investigating How Well NDec Works in People With Sickle Cell Disease (PHASE2)
- Combination of Thalidomide and Hydroxyuria in Transfusion Dependent Thalasemmia (PHASE2)
- A Phase 1/2 Study of CPI-0610 With and Without Ruxolitinib in Patients With Hematologic and Myeloproliferative Malignancies (PHASE1, PHASE2)
- A Phase 1 Study of OTS-412 (Recombinant Oncolytic Vaccinia Virus) in Treatment-refractory Solid Tumor Patients (PHASE1)
- A Phase 1 Study of OTS-412 (Recombinant Oncolytic Vaccinia Virus) in Treatment-refractory Solid Tumor Patients (South Korea) (PHASE1)
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 (HU) CI brief — competitive landscape report
- Hydroxyurea (HU) updates RSS · CI watch RSS
- Bionoxx Inc. portfolio CI