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Hydroxycarbamide

University of Birmingham · Phase 3 active Small molecule Under review

Hydroxycarbamide is a Small molecule drug developed by University of Birmingham. It is currently in Phase 3 development for Chronic myeloid leukemia, Hb SS disease, Hemoglobin SS disease with crisis. Also known as: Hydroxyurea, Siklos.

Hydroxycarbamide, also known as hydroxyurea, is a small molecule inhibitor of ribonucleoside-diphosphate reductase RR1, used to treat various conditions including sickle-cell disease, where it increases fetal hemoglobin and decreases the number of attacks. It is taken by mouth and has been studied in clinical trials for other conditions such as intracranial meningioma, idiopathic pulmonary fibrosis, and emphysema or COPD.

Likelihood of approval
61.3% vs 58.3% industry baseline
If approved by FDA: likely 2028–2030
Steps remaining: NDA/BLA submission
Confidence: High
Why this estimate
  • 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.
Predicted approval windows by jurisdiction (conditional on FDA approval)
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 nameHydroxycarbamide
Also known asHydroxyurea, Siklos
SponsorUniversity of Birmingham
TargetCarbonic anhydrase 2, Carbonic anhydrase 5A, mitochondrial, Carbonic anhydrase 9
ModalitySmall molecule
Therapeutic areaOncology
PhasePhase 3

Approved indications

Common side effects

No common side effects on file.

Key clinical trials

Primary sources

Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.

SourceUsed for
ClinicalTrials.govTrial 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:

Frequently asked questions about Hydroxycarbamide

What is Hydroxycarbamide?

Hydroxycarbamide is a Small molecule drug developed by University of Birmingham, indicated for Chronic myeloid leukemia, Hb SS disease, Hemoglobin SS disease with crisis.

What is Hydroxycarbamide used for?

Hydroxycarbamide is indicated for Chronic myeloid leukemia, Hb SS disease, Hemoglobin SS disease with crisis, Malignant melanoma, Malignant tumor of head and/or neck.

Who makes Hydroxycarbamide?

Hydroxycarbamide is developed by University of Birmingham (see full University of Birmingham pipeline at /company/university-of-birmingham).

Is Hydroxycarbamide also known as anything else?

Hydroxycarbamide is also known as Hydroxyurea, Siklos.

What development phase is Hydroxycarbamide in?

Hydroxycarbamide is in Phase 3.

What does Hydroxycarbamide target?

Hydroxycarbamide targets Carbonic anhydrase 2, Carbonic anhydrase 5A, mitochondrial, Carbonic anhydrase 9.

Related

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing