Sickle cell anemia is an inherited blood disorder resulting from mutations in the beta-globin gene. This genetic mutation leads to the creation of abnormal hemoglobin, which causes red blood cells to take on a sickle shape. The sickle cells can block blood flow, leading to severe pain, organ damage, and even death. Voxelotor, also known by its brand name Oxbryta, is a newly approved FDA medication that increases hemoglobin affinity for oxygen, reducing the formation of sickle cells in patients with sickle cell anemia.
Chemical Name: (R)-3-(4-(1-(Cyclopropylmethyl)-1H-pyrazol-5-yl)-3-fluorophenyl)-5-fluoro-1-(2-fluorophenyl)-1H-pyrazole-4-carboxamide.
Molecular formula: C19H12F3N5O
Formula weight: 401.3 g/mol
CAS No: 1446321-46-5
Top Ten Keywords from Google and Synonyms
Voxelotor Synonyms: Oxbryta, GBT440, FT-4202, VYN-445303
Sickle Cell Anemia Synonyms: Sickle cell disease, hemoglobin SS disease, sickling disorder.
Hemoglobin Affinity Synonyms: Oxygen Affinity, Hemoglobin-Oxygen Dissociation Curve
Clinical trial: Synonyms: Medical trial, Clinical study, Medical research
FDA Approval Synonyms: US Food and Drug Administration, Regulatory agency
Hydroxyurea Synonyms: Hydrea, Droxia
Red Blood Cells Synonyms: RBCs, Erythrocytes
Oxygen Saturation Synonyms: O2 saturation, arterial oxygen saturation
Hydroxyurea resistance Synonyms: Hydroxyurea treatment failure, inadequate response to hydroxyurea
Anemia Synonyms: Iron-deficiency anemia, Sickle cell anemia, Hemolytic anemia, Aplastic anemia.
Health Benefits of Voxelotor
Voxelotor is the first FDA-approved therapy specifically developed to target the underlying cause of sickle cell anemia. It works by binding to hemoglobin, a protein in red blood cells that carries oxygen throughout the body. In normal blood, hemoglobin molecules carry oxygen from the lungs to the body tissues, and then carry carbon dioxide from the tissues back to the lungs, where it is exhaled. However, in sickle cell anemia, the abnormal hemoglobin causes red blood cells to become stiff and misshapen, leading to blockages in blood vessels and impaired blood flow.
Voxelotor enhances the affinity of hemoglobin for oxygen, making it more efficient at carrying oxygen and reducing the formation of sickle-shaped red blood cells. In clinical trials, voxelotor demonstrated significant reductions in hemolysis, the breakdown of red blood cells, and improved markers of anemia, including hemoglobin levels and reticulocyte counts.
Potential Effects
Historically, hydroxyurea has been the only FDA-approved medication for the management of sickle cell anemia. However, despite its clinical benefits, hydroxyurea is not effective for all patients and is associated with numerous adverse effects. Voxelotor is a novel medication that offers an alternative treatment option for sickle cell anemia that does not involve cytotoxicity
