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Hydroxyurea treatment (HU)
Hydroxyurea treatment (HU) is a Ribonucleotide reductase inhibitor Small molecule drug developed by Assistance Publique - Hôpitaux de Paris. It is currently in Phase 3 development for Sickle cell disease, Polycythemia vera, Essential thrombocythemia.
Hydroxyurea inhibits ribonucleotide reductase to reduce DNA synthesis and increase fetal hemoglobin production, reducing sickling and hemolysis.
Hydroxyurea inhibits ribonucleotide reductase to reduce DNA synthesis and increase fetal hemoglobin production, reducing sickling and hemolysis. Used for Sickle cell disease, Polycythemia vera, Essential thrombocythemia.
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Baseline phase 3 → approval rate
+58.3pp
Industry-wide phase 3 drugs reach approval ~58.3% of the time (BIO/Informa 2023 industry benchmark across all therapeutic areas). -
Oncology Phase 3 boost
+3.0pp
Oncology Phase 3 trials have higher approval rates (~61%) than the cross-industry average due to clearer endpoints and FDA oncology pathway.
| Regulator | Country | Likely year | Lag vs FDA |
|---|---|---|---|
| FDA | US | 2028–2030 | — |
| EMA | EU | 2029–2031 | +0.7 yr |
| MHRA | GB | 2029–2031 | +0.7 yr |
| Health Canada | CA | 2029–2032 | +0.9 yr |
| TGA | AU | 2029–2032 | +1.2 yr |
| PMDA | JP | 2029–2032 | +1.5 yr |
| NMPA | CN | 2030–2033 | +2.3 yr |
| MFDS | KR | 2029–2032 | +1.4 yr |
| CDSCO | IN | 2029–2033 | +1.8 yr |
| ANVISA | BR | 2030–2033 | +2.3 yr |
Hover any row for the lag rationale. Lag estimates are reduced when the drug has FDA Breakthrough or EMA PRIME designation (sponsors file globally in parallel).
Estimate based on the BIO/Informa industry phase transition rates plus per-drug modifiers for therapeutic area, sponsor type, FDA designations, mechanism, and trial design. Per-jurisdiction lags from Tufts CSDD international approval studies. Not investment, clinical or regulatory advice. Methodology: /methodology#likelihood.
At a glance
| Generic name | Hydroxyurea treatment (HU) |
|---|---|
| Sponsor | Assistance Publique - Hôpitaux de Paris |
| 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 decreases deoxyribonucleotide pools, slowing DNA replication and cell division. In sickle cell disease, it increases fetal hemoglobin (HbF) expression, which does not polymerize and prevents red blood cell sickling. This reduces vaso-occlusive crises, hemolytic anemia, and organ damage.
Approved indications
- Sickle cell disease
- Polycythemia vera
- Essential thrombocythemia
- Chronic myeloid leukemia
Common side effects
- Myelosuppression (anemia, leukopenia, thrombocytopenia)
- Macrocytosis
- Nausea and vomiting
- Hyperuricemia
- Elevated creatinine
Key clinical trials
- Evaluation of HU-resistance in Adult Patients With Polycythemia Vera Who Meet PV-AIM Predictors (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)
- T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias (PHASE1, PHASE2)
- A Study to Access Efficacy and Safety of P1101 in Chinese PV Patients Who Are Intolerant or Resistance to HU (PHASE2)
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 treatment (HU) CI brief — competitive landscape report
- Hydroxyurea treatment (HU) updates RSS · CI watch RSS
- Assistance Publique - Hôpitaux de Paris portfolio CI
Frequently asked questions about Hydroxyurea treatment (HU)
What is Hydroxyurea treatment (HU)?
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Related
- Drug class: All Ribonucleotide reductase inhibitor drugs
- Target: All drugs targeting Ribonucleotide reductase
- Manufacturer: Assistance Publique - Hôpitaux de Paris — full pipeline
- Therapeutic area: All drugs in Hematology/Oncology
- Indication: Drugs for Sickle cell disease
- Indication: Drugs for Polycythemia vera
- Indication: Drugs for Essential thrombocythemia
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing