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hydroxyurea + sildenafil
- Development stage
- Phase 1
- Lead developer
- National Heart
- Modality
- Small Molecules
- Administration
- Oral
01
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.
02
Targets
PDE5 (Phosphodiesterase 5A)RNR (Ribonucleotide reductase)
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