Drug intelligence / Profile preview

hydroxycarbamide + magnesium pidolate

Development stage
Phase 2
Modality
Small Molecules
Administration
Oral
01

Overview

This is a combination of two drugs, hydroxycarbamide (also known as hydroxyurea) and magnesium pidolate, investigated primarily for the treatment of sickle cell disease. Hydroxycarbamide is an antimetabolite that increases fetal hemoglobin (HbF) levels and reduces sickling in red blood cells by inhibiting DNA synthesis. Magnesium pidolate acts to increase intracellular magnesium in erythrocytes, which helps reduce red cell dehydration by negatively regulating membrane transporters involved in water loss. The rationale for combining these agents is their distinct mechanisms—hydroxycarbamide increases HbF while magnesium pidolate addresses cellular dehydration—potentially offering additive or synergistic effects against sickling crises[1][3][2]. Clinical studies have focused on determining the maximum tolerated dose and safety profile of this combination in children with sickle cell anemia[1].

Other names
hydroxyurea + magnesium pidolate
02

Targets

RNR (Ribonucleotide reductase)HbF (Fetal hemoglobin)

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