EU approves Oxbryta for hemolytic anemia due to sickle cell disease – Global Blood Therapeutics

Global Blood Therapeutics announced the European Commission (EC) has granted 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 as monotherapy or in combination with hydroxycarbamide (hydroxyurea). Oxbryta, a once-daily, oral treatment, is the first medicine approved in Europe that directly inhibits sickle hemoglobin (HbS) polymerization, the molecular basis of sickling and destruction of red blood cells in SCD.

The EC approval follows a positive opinion by the Committee for Medicinal Products for Human Use (CHMP) in December 2021 based on results of the Phase III HOPE (Hemoglobin Oxygen Affinity Modulation to Inhibit HbS PolymErization) Study demonstrating clinically meaningful and statistically significant improvements in hemoglobin (Hb) levels, accompanied by hemolysis, for patients treated with Oxbryta. Results from the HOPE Study were published in June 2019 in The New England Journal of Medicine and the analysis of the complete data from the HOPE Study were published in The Lancet Haematology in April 2021.

In the HOPE Study, Oxbryta demonstrated a favorable safety profile with limited and transitory adverse reactions. The most common adverse reactions occurring in at least 10% of patients treated with Oxbryta (1500mg) with a difference of >3% compared to placebo were headache (26% vs. 22%), diarrhea (20% vs. 10%), abdominal pain (14% vs. 8%), nausea (17% vs. 10%), fatigue (14% vs. 10%), rash (14% vs. 10%) and pyrexia (12% vs. 7%).