Abstract

Sickle cell disease is one challenging blood disorder, affecting around 100,000 people in the United States alone. None of the currently approved drugs can modify the underlying pathology of the disease. Voxelotor, first of its kind, is an orally administered drug that can alter the underlying disease pathology (by increasing the affinity between Hb and oxygen) and inhibit sickling of red blood cells. Several clinical trials and case series have documented the benefits and safety of voxelotor therapy in sickle cell disease. Currently, the US FDA has approved the drug for treatment of sickle cell disease and also granted the status of orphan drug.

MeSH

Pathology, Pharmaceutical Preparations, Cells, Therapeutics, Blood

Cited by

• The Efficacy of Marijuana Use for Pain Relief in Adults With Sickle Cell Disease: A Systematic Review. Cureus, 2022
• Roles of Pharmacists in the Management of Sickle Cell Disease in Adults: A Narrative Review. J Pharm Technol, 2024
• Genome-based therapeutic interventions for β-type hemoglobinopathies. Hum Genomics, 2022
• A retrospective research of adverse event reporting system events for voxelotor based on the FAERS database. BMC Pharmacol Toxicol, 2025
• Computational screening of phytochemicals present in some Nigerian medicinal plants against sickle cell disease. Sci Rep, 2024
• Hydroxyurea and blood transfusion therapy for Sickle cell disease in South Asia: inconsistent treatment of a neglected disease. Orphanet J Rare Dis, 2021
• Complement in Sickle Cell Disease: Are We Ready for Prime Time? J Blood Med, 2021
• More to voxelotor than meets the eye? Blood Adv, 2022