Oxbryta®: 1st Therapy Approved in European Union to Target Cause of Sickle Cell Disease

Global Blood Therapeutics, Inc. (GBT) announced the approve of Marketing Authorization for Oxbryta® (voxelotor) for the treatment of hemolytic anemia due to sickle cell disease (SCD) in adult and pediatric patients 12 years of age and older from the European Commission (EC). Oxbryta, given as a once-daily oral treatment, is the first medicine approved in Europe that directly suppresses the sickle hemoglobin (HbS) polymerization, the molecular basis of sickling and destruction of red blood cells in SCD. Eligible patients may take the therapy as monotherapy or in combination with hydroxycarbamide (hydroxyurea), a standard treatment to reduce the frequency of vaso-occlusive crises — episodes of acute pain caused by sickled red blood cells blocking blood flow — and the need for blood transfusions. Oxbryta demonstrated a favorable safety profile with limited and transitory adverse events compared with placebo groups. For more information: Global Blood Therapeutics, press release.