Voxelotor


Voxelotor, sold under the brand name Oxbryta, is a drug for the treatment of sickle cell disease. Developed by Global Blood Therapeutics, voxelotor is the first hemoglobin oxygen-affinity modulator. Voxelotor has been shown to have disease-modifying potential by increasing hemoglobin levels and decreasing hemolysis indicators in sickle cell patients. It has a safe profile in sickle cell patients and healthy volunteers, without any dose-limiting toxicity.
In November 2019, voxelotor received accelerated approval in the United States for the treatment of sickle cell disease for those 12 years of age and older.

Side effects

Common side effects for people taking voxelotor were headache, diarrhea, abdominal pain, nausea, fatigue, rash and pyrexia.

History

Voxelotor was granted accelerated approval by the U.S. Food and Drug Administration in November 2019, and further clinical trials are required to verify and describe Oxbryta's clinical benefit. The FDA granted the application for voxelotor fast track designation and orphan drug designation.
The approval of voxelotor was based on the results of a clinical trial with 274 subjects with sickle cell disease.
The FDA granted the approval of Oxbryta to Global Blood Therapeutics.