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Hydroxycarbamid
Hydroxycarbamid is a Small molecule drug developed by Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts. It is currently in Phase 2 development for Chronic myeloid leukemia, Hb SS disease, Hemoglobin SS disease with crisis. Also known as: siklos, Hydroxyurea.
Hydroxycarbamide, also known as hydroxyurea, is a small molecule inhibitor of the enzyme ribonucleoside-diphosphate reductase RR1. It has been studied in clinical trials for various conditions, including sickle cell anemia, myelofibrosis, leukemia, HIV infections, and sickle cell disease.
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Baseline phase 2 → approval rate
+15.3pp
Industry-wide phase 2 drugs reach approval ~15.3% of the time (BIO/Informa 2023 industry benchmark across all therapeutic areas). -
Oncology Phase 2 attrition
-2.0pp
Oncology drugs have higher Phase 2-to-Phase 3 attrition than average — many fail to show OS benefit in larger studies.
| Regulator | Country | Likely year | Lag vs FDA |
|---|---|---|---|
| FDA | US | 2031–2034 | — |
| EMA | EU | 2032–2035 | +0.7 yr |
| MHRA | GB | 2032–2035 | +0.7 yr |
| Health Canada | CA | 2032–2036 | +0.9 yr |
| TGA | AU | 2032–2036 | +1.2 yr |
| PMDA | JP | 2032–2036 | +1.5 yr |
| NMPA | CN | 2033–2037 | +2.3 yr |
| MFDS | KR | 2032–2036 | +1.4 yr |
| CDSCO | IN | 2032–2037 | +1.8 yr |
| ANVISA | BR | 2033–2037 | +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 | Hydroxycarbamid |
|---|---|
| Also known as | siklos, Hydroxyurea |
| Sponsor | Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts |
| Target | Carbonic anhydrase 2, Carbonic anhydrase 5A, mitochondrial, Carbonic anhydrase 9 |
| Modality | Small molecule |
| Therapeutic area | Oncology |
| Phase | Phase 2 |
Approved indications
- Chronic myeloid leukemia
- Hb SS disease
- Hemoglobin SS disease with crisis
- Malignant melanoma
- Malignant tumor of head and/or neck
- Malignant tumor of ovary
- Sickling disorder due to hemoglobin S
Common side effects
Key clinical trials
- Bomedemstat vs Hydroxyurea for Essential Thrombocythemia (MK-3543-007) (PHASE3)
- A Study to Investigate the Efficacy and Safety of Crizanlizumab (5 mg/kg) Compared With Placebo in Adolescent and Adult Sickle Cell Disease Patients Who Experience Frequent Vaso-Occlusive Crises (SPARKLE) (PHASE3)
- Campath/Fludarabine/Melphalan Transplant Conditioning for Non-Malignant Diseases (PHASE1, PHASE2)
- Study on Efficacy and Safety of Givinostat Versus Hydroxyurea in Patients With Polycythemia Vera (PHASE3)
- Genetically Engineered Cells (CD83 CAR T Cells) for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia (PHASE1)
- Comparing the Radiopharmaceutical Drug, [177Lu]Lu-DOTATATE, to Standard of Care Treatment for Patients With Meningioma That Has Come Back After Prior Treatment (PHASE2)
- Integrating Point of Care Testing (POCT) For Newborn Screening and Early Care for Sickle Cell Disease in Yopougon, Côte d'Ivoire (PHASE4)
- A Study of Bomedemstat (IMG-7289/MK-3543) Compared to Best Available Therapy (BAT) in Participants With Essential Thrombocythemia and an Inadequate Response or Intolerance of Hydroxyurea (MK-3543-006) (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:
- Hydroxycarbamid CI brief — competitive landscape report
- Hydroxycarbamid updates RSS · CI watch RSS
- Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts portfolio CI
Frequently asked questions about Hydroxycarbamid
What is Hydroxycarbamid?
What is Hydroxycarbamid used for?
Who makes Hydroxycarbamid?
Is Hydroxycarbamid also known as anything else?
What development phase is Hydroxycarbamid in?
What does Hydroxycarbamid target?
Related
- Target: All drugs targeting Carbonic anhydrase 2, Carbonic anhydrase 5A, mitochondrial, Carbonic anhydrase 9
- Manufacturer: Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts — full pipeline
- Therapeutic area: All drugs in Oncology
- Indication: Drugs for Chronic myeloid leukemia
- Indication: Drugs for Hb SS disease
- Indication: Drugs for Hemoglobin SS disease with crisis
- Also known as: siklos, Hydroxyurea
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing