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hydroxyurea
- Development stage
- Approved
- Lead developer
- Bristol Myers Squibb
- Modality
- Orthosteric Ligands → Classical Binding Small Molecules → Small Molecules, Metabolically Activated Prodrugs → Prodrugs/Conditional Activator Small Molecules → Small Molecules, Nucleic Acid-Directed Small Molecules → Small Molecules
- Administration
- Oral
01
Overview
Hydroxyurea (also known as hydroxycarbamide) is an oral small molecule antimetabolite used primarily in the treatment of sickle cell disease and various myeloproliferative neoplasms such as polycythemia vera (PV), essential thrombocythemia (ET), and chronic myeloid leukemia (CML). It is also indicated for certain head and neck cancers and cervical cancer. Hydroxyurea works by inhibiting ribonucleotide reductase through scavenging tyrosyl free radicals, thereby blocking the synthesis of deoxyribonucleotides required for DNA replication. In sickle cell disease, it increases fetal hemoglobin levels via mechanisms that may involve nitric oxide-mediated activation of guanylate cyclase and increased gamma-globin synthesis. This reduces vaso-occlusive crises and the need for blood transfusions[1][2][5][7]. Hydroxyurea is included on the World Health Organization's List of Essential Medicines.
- Brand names
- HydreaSiklosMylocelDroxiaXromiAPO-HYDROXYUREAGEN-HYDROXYUREA
- Other names
- 羟基脲
02
Targets
RNR (Ribonucleotide reductase)
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