Voxelotor, a drug for sickle cell disease

Name: Voxelotor

CAS: 1446321-46-5

Originating company: Global Blood Therapeutics

Indication: Sickle cell disease

Time on Market
The FDA authorized Oxbryta (voxelotor) for the treatment of sickle cell disease (SCD) in adults and children patients 12 years of age and older on November 25, 2019.The European Commission (EC) approved the sale of Oxbryta (voxelotor) on February 16, 2022, for the treatment of sickle cell disease (SCD) in adults and pediatric patients 12 years of age and older, as well as for the treatment of hemolytic anemia brought on by SCD, either as a monotherapy or in combination with hydroxyurea. China, Japan, etc. don't yet have it available.

Sickle Cell Disease
A collection of genetic red blood cell diseases is known as sickle cell disease (SCD). Hemoglobin is an oxygen-carrying protein found in red blood cells. Round and healthy red blood cells use tiny blood capillaries to transport oxygen to different parts of the body. Red blood cells in persons with SCD have defective hemoglobin, which makes them hard and sticky and resemble the "sickle"-shaped farm implement. Red blood cells are constantly in insufficient supply due to the premature death of sickle cells. Additionally, they become caught and obstruct blood flow when they cross tiny blood veins. This may result in discomfort and other severe side effects, including infections, acute chest syndrome, and strokes.