FDA Approves Global Blood Therapeutics’ Sickle Cell Disease Treatment

Global Blood Therapeutics received accelerated approval for Oxbryta (voxelotor) to treat sickle cell disease in adults and pediatric patients 12 years of age and older.

The approval was based on the results of a clinical trial involving 274 sickle cell patients given different doses of the drug. The study showed a 51.1 percent increase in hemoglobin response rate compared to 6.5 percent in the placebo group.

The FDA handed the medication breakthrough therapy, fast track, orphan drug, and rare pediatric disease designations, as well as priority review.