Drug intelligence / Profile preview

deferasirox + hydroxyurea

Development stage
Preclinical
Lead developer
Novartis
Modality
Small Molecules
Administration
Oral
01

Overview

Deferasirox + hydroxyurea is a combination therapy used primarily in patients with sickle cell disease who require both iron chelation and disease-modifying treatment. Deferasirox is an oral iron chelator that binds trivalent (ferric) iron, forming a stable complex that is excreted from the body, thereby reducing chronic iron overload resulting from repeated blood transfusions[9][5]. Hydroxyurea is an antineoplastic agent that inhibits ribonucleotide reductase, leading to decreased DNA synthesis and increased fetal hemoglobin (HbF) production, which reduces the frequency of vaso-occlusive crises in sickle cell disease[6][10]. The concurrent use of these two drugs addresses both the complications of chronic transfusion therapy (iron overload) and the underlying pathophysiology of sickle cell disease. Studies indicate that three months of concurrent use appears safe, but further research on long-term safety and efficacy is needed[1][2].

02

Targets

RRM1RRM2

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