Drug intelligence / Profile preview

hydroxyurea + sildenafil

Development stage: Unknown
Lead developer: National Heart
Modality: Small Molecules
Administration: Oral

Overview

A combination therapy consisting of hydroxyurea and sildenafil used in the treatment of sickle cell anemia. Hydroxyurea works by inhibiting ribonucleotide reductase, blocking DNA synthesis, and inducing fetal hemoglobin (HbF) production through nitric oxide-mediated mechanisms. Sildenafil, a phosphodiesterase 5 inhibitor, potentiates cGMP-dependent signaling and complements hydroxyurea therapy by improving hemodynamic status. Clinical studies have shown that this combination decreased pulmonary artery systolic pressure and increased exercise capacity in sickle cell disease patients.

Targets

RNR (Ribonucleotide reductase)PDE5 (Phosphodiesterase 5A)

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