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Hydroxyurea (hydroxycarbamide)

Hydroxyurea is a cheap, decades-old pill that lowers high blood counts in polycythaemia vera and essential thrombocythaemia and is also the main drug for sickle cell disease.

First-line cytoreduction in high-risk PV and ET (reduces thrombosis; PT-1 trial superior to anagrelide in ET). Historic role in CML before imatinib. Also used in sickle cell disease and, less often, for leukocytosis in acute leukaemia. Concerns: leg ulcers, skin cancers, questionable leukaemogenicity.

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Hydroxyurea
PubChem
Modality
Oral ribonucleotide reductase inhibitor (cytoreductive)
Mechanism
Inhibits ribonucleotide reductase, depleting deoxyribonucleotides and arresting S-phase; lowers blood counts in myeloproliferative disease.
Brand / code
Hydrea / Droxia / Siklos
Medicare
Part D (self-administered)

Oral, self-administered, so it is a Part D drug: covered through a stand-alone Part D plan or Medicare Advantage drug benefit, usually on the specialty tier with 25 to 33% coinsurance until the annual cap ($2,000 in 2025, $2,100 in 2026). Generic; typically a low-tier drug costing a few dollars a month.

Commercial insurance
covered on label

Multi-source generic on low-cost tiers; usually no prior authorisation. Cash prices without insurance are modest.

Sources: Medicare.gov: Drug coverage (Part D) · Medicare.gov: Costs for Medicare drug coverage (annual out-of-pocket cap). Not medical or financial advice; verify with your plan.

Not appraisedSearch NICE
Notes
Generic; first-line cytoreduction in polycythaemia vera and essential thrombocythaemia and used in CML and sickle cell disease. Funded routinely.
NHS England
Routinely funded for the appraised indication (or via managed access)

Sources: NICE search: hydroxycarbamide. Funding decisions are indication-specific and change monthly; verify with NICE and your treating team.

Regulatory

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Approvals

RegionYearIndication
US1967CML, head and neck cancer with radiotherapy, melanoma, ovarian cancer (historic labelling)
US1998Sickle cell anaemia (Droxia)

Trials

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ClinicalTrials.gov · phase 2/3
refreshed 2026-09-09
214 studies11 recruiting75 Phase 235 Phase 3
Search “Hydroxyurea” on ClinicalTrials.gov →
Counts are from a name search and may include unrelated studies; up to 100 studies are summarised.

Recruiting now (live from ClinicalTrials.gov)

Recruiting trials near you · live from ClinicalTrials.gov
Hydroxyurea (hydroxycarbamide)
intervention: Hydroxyurea
Open on ClinicalTrials.gov →

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Latest papers

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Latest papers · live from Europe PMC
Open in Europe PMC

Query for this drug: (TITLE:"Hydroxyurea" OR ABSTRACT:"Hydroxyurea" OR TITLE:"hydroxycarbamide" OR ABSTRACT:"hydroxycarbamide" OR TITLE:"Hydrea" OR ABSTRACT:"Hydrea" OR TITLE:"Droxia" OR ABSTRACT:"Droxia" OR TITLE:"Siklos" OR ABSTRACT:"Siklos") AND (cancer OR tumor OR tumour OR oncology OR carcinoma OR lymphoma OR leukemia OR leukaemia OR myeloma OR sarcoma OR melanoma OR glioma). Results are unfiltered search hits about Hydroxyurea (hydroxycarbamide), not a curated reading list.

Connected

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