Hydroxyurea
- Generic Name
- Hydroxyurea
- Brand Names
- Droxia, Hydrea, Siklos, Xromi
- Drug Type
- Small Molecule
- Chemical Formula
- CH4N2O2
- CAS Number
- 127-07-1
- Unique Ingredient Identifier
- X6Q56QN5QC
- Background
An antineoplastic agent that inhibits DNA synthesis through the inhibition of ribonucleoside diphosphate reductase.
- Indication
Hydroxyurea is indicated to reduce the frequency of painful crises and to reduce the need for blood transfusions in adult and pediatric patients, 2 years of age and older, with sickle cell anemia with recurrent moderate to severe painful crises.
- Associated Conditions
- Chronic Myelogenous Leukemia (CML), Essential Thrombocythemia (ET), Head and Neck Primary Squamous Cell Carcinoma, Hypereosinophilic Syndrome (HES), Locally Advanced Squamous Cell Carcinomas of the Head and Neck (SCCHN), Meningiomas, Polycythemia Vera (PV), Sickle Cell Crisis, Vaso-occlusive Crisis
- Associated Therapies
- Chemoradiotherapy, Radiation Therapy
Expanding Treatment Horizons for Sickle Cell Disease: Barriers, Innovations, and Future Directions
Sickle cell disease (SCD) affects 20 million globally, with standard treatments including hydroxyurea, voxelotor, pain management, and BMT. Haplo-HSCT and gene therapies offer alternatives when MSD BMTs are unavailable. SCD causes chronic hemolysis, inflammation, and premature death, with supportive care aiming to reduce disease progression. BMT is effective but has complications like GVHD, and barriers include donor availability and financial constraints. Gene therapy shows promise with reduced SCD-related complications but faces issues like cost and accessibility. Haplo-HSCT, with improved responses in trials, may serve as an alternative.
Merck's Bomedemstat Begins Phase 3 Trial for Essential Thrombocythemia
Merck launched Shorespan-007, a Phase 3 trial evaluating bomedemstat for essential thrombocythemia (ET) patients not on cytoreductive therapy. Bomedemstat is compared to hydroxyurea in 300 ET patients, with primary and secondary endpoints focusing on response rates and symptom scores. Bomedemstat also has FDA Orphan Drug and Fast Track designations for ET and myelofibrosis, and a Priority Medicines designation for myelofibrosis by the EMA.
Merck Launches Shorespan-007 Clinical Trial of Bomedemstat for Essential Thrombocythemia
Merck's Phase III Shorespan-007 trial evaluates bomedemstat for essential thrombocythemia (ET) in patients not previously treated with cytoreductive therapy, comparing it to hydroxyurea. The trial aims to improve disease control and quality of life for ET patients, with endpoints including durable clinicohematologic response rate and patient-reported outcomes. Bomedemstat has received Orphan Drug and Fast Track designations for ET and myelofibrosis.
Merck Announces Phase 3 Trial Initiation for Bomedemstat, an Investigational Candidate for ...
Merck initiates Shorespan-007, a Phase 3 trial evaluating bomedemstat for essential thrombocythemia (ET) patients not previously treated with cytoreductive therapy, aiming to address unmet needs in myeloproliferative neoplasms (MPNs).
Merck Announces Phase 3 Trial Initiation for Bomedemstat, an Investigational Candidate for the Treatment of Certain Patients With Essential Thrombocythemia
Merck initiates Phase 3 trial, Shorespan-007, evaluating bomedemstat, an LSD1 inhibitor, for treating essential thrombocythemia (ET) patients not previously treated with cytoreductive therapy. The trial aims to compare bomedemstat with standard care chemotherapy, hydroxyurea, with a focus on durable clinicohematologic response rate and patient-reported outcomes. Bomedemstat holds FDA Orphan Drug and Fast Track Designations for ET and myelofibrosis.
Drug Development Updates
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