Jonathan Sorof, MD of Global Blood Therapeutics (GBT) explains the mechanism of action of Oxbryta (voxelotor) to treat persons with sickle cell disease.
Voxelotor blocks deoxygenated sickle hemoglobin polymerization and attenuates red blood cells from ‘sickling’.
Sickle cell disease is a rare genetic blood disorder in which red blood cells are abnormally shaped and ‘sticky’, which restricts blood flow. The condition can lead to severe pain and organ damage. It affects approximately 100,000 people in the United States.
Voxelotor was given accelerated approvsl by the U.S. Food and Drug Administration in November of 2019 to treat persons, 12 years and older, with sickle cell disease.
To learn more about Sickle Cell Disease and other rare blood disorders, visit checkrare.com/diseases/