On Monday, the U.S. Food and Drug Administration (FDA) announced accelerated approval of Oxbryta (voxelotor) for the treatment of sickle cell disease in patients aged 12 and above.
“Oxbryta is the first approved treatment for sickle cell disease that directly inhibits the root cause of the disease, sickle hemoglobin polymerization.”
Human trials were conducted on 274 patients with sickle cell disease, of which, 90 patients were given 1,500 mg Oxbryta, 92 patients were given 900 mg, while the remaining 92 patients were given a placebo.
After 24 weeks, more than 51 percent of patients who received Oxbryta 1,500 mg had a greater hemoglobin response when compared with only 6.5 percent of patients who were given a placebo.
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