osivelotor

Osivelotor is an orally administered small molecule in development for the treatment of sickle cell disease (SCD). It acts as a next-generation sickle hemoglobin polymerization inhibitor, similar in mechanism to voxelotor but with improved pharmacokinetic properties. Osivelotor binds directly to hemoglobin, increasing its affinity for oxygen and stabilizing it in the oxygenated state. This prevents deoxygenated sickle hemoglobin (HbS) from polymerizing, thereby reducing red blood cell sickling and improving red blood cell health. Clinical trials have shown that osivelotor increases hemoglobin levels, improves markers of hemolysis, enhances red blood cell deformability, and reduces vaso-occlusive crises in SCD patients. The drug is being developed by Pfizer and is currently undergoing Phase 2/3 clinical trials for SCD[1][4][5][6][8][9].

Brand names

osivelotor

Other names

(S)-2-hydroxy-6-((4-(2-(2-hydroxyethyl)nicotinoyl)morpholin-3-yl)methoxy)benzaldehyde