Oxbryta filed and validated by MAA for sickle cell disease.- Global Blood Therapeutics

Global Blood Therapeutics announced that the European Medicines Agency (EMA) has completed the validation of GBT’s Marketing Authorization Application (MAA) for Oxbryta (voxelotor) tablets and started its standard review process. GBT is seeking full marketing approval from the EMA for Oxbryta to treat hemolytic anemia in patients with sickle cell disease (SCD) who are 12 years of age and older.The MAA is based on data from the Phase III HOPE (Hemoglobin Oxygen Affinity Modulation to Inhibit HbS PolymErization) Study and the Phase II HOPE-KIDS 1 Study, both of which enrolled patients at clinical sites in Europe. The HOPE Study achieved its primary endpoint of an improvement in hemoglobin (Hb) levels of greater than 1 g/dL at 24 weeks with significant improvements in markers of hemolysis in indirect bilirubin and reticulocyte percentage. The most common side effects reported in the HOPE Study at 24 weeks occurring in at least 10% of patients treated with Oxbryta with a difference of more than 3% compared to placebo were headache (26% vs. 22%), diarrhea (20% vs. 10%), abdominal pain (19% vs. 13%), nausea (17% vs. 10%), fatigue (14% vs. 10%), rash (14% vs. 10%) and pyrexia (12% vs. 7%). The analysis of the complete data from the HOPE Study further demonstrated that Oxbryta, at a daily dose of 1500 mg, resulted in durable improvements in Hb levels and markers of hemolysis over 72 weeks of treatment in SCD patients 12 years of age and older. Treatment with Oxbryta was well tolerated, with no new safety or tolerability issues identified. The 72-week results from the HOPE Study were presented at the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition in December 2020.