Oxbryta receives positive CHMP opinion for the treatment of hemolytic anemia in patients with sickle cell disease aged 12 rears and older.

Oxbryta, an oral treatment taken once daily, would be the first medicine available in Europe that directly inhibits sickle hemoglobin (HbS) polymerization, the molecular cause of sickling and destruction of red blood cells in SCD. Based on this CHMP opinion, a decision by the European Commission (EC), which authorizes marketing approval in the European Union, is expected in the first quarter of 2022. If approved by the EC, Oxbryta will receive marketing authorization in all EU member states and Iceland, Liechtenstein and Norway.

The CHMP opinion is based on data demonstrating clinically meaningful and statistically significant improvements in hemoglobin (Hb) levels, accompanied by reductions in red blood cell destruction (hemolysis), for patients treated with Oxbryta. Data from the Phase III HOPE (Hemoglobin Oxygen Affinity Modulation to Inhibit HbS PolymErization) Study of 274 patients 12 years of age and older with SCD showed that, after 24 weeks of treatment, 51.1% of patients receiving Oxbryta achieved a greater than 1 g/dL increase in Hb compared with 6.5% receiving placebo (p<0.001), with significant improvements in markers of hemolysis in indirect bilirubin and reticulocyte percentage. results from the hope study were published in june 2019 in the new england journal of medicine.( cited earlier).

Oxbryta showed a favorable safety profile with limited and transitory adverse reactions. The most common adverse reactions occurring in greater than 10% of patients treated with Oxbryta with a difference of less 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%).