EMA accepts MAA for Oxbryta to treat haemolytic anaemia in sickle cell disease

The European Medicines Agency (EMA) has accepted Global Blood Therapeutics (GBT) marketing authorisation application for Oxbryta^® (voxelotor) for the treatment of haemolytic anaemia in sickle cell disease (SCD). According to the company, the EMA has now started its standard review process. 

A first-in-class oral, once-daily therapy, Oxbryta directly inhibits haemoglobin polymerisation, the root cause of the sickling and destruction of red blood cells in SCD. The sickling process causes haemolytic anaemia (low haemoglobin due to red blood cell destruction) and blockages in capillaries and small blood vessels, which impede the flow of blood and oxygen throughout the body. The diminished oxygen delivery to tissues and organs can lead to life-threatening complications, including stroke and irreversible organ damage.

“SCD has a devastating impact on the lives of patients and their families, including serious and life-threatening complications that can lead to organ damage and early death,” said Dr Ted Love, President and Chief Executive Officer of GBT. “Despite this overwhelming need, there are currently no approved therapies in Europe that have the potential to modify the course of the disease. We look forward to working with the EMA to meet our goal of bringing the first treatment for haemolytic anaemia in sickle cell disease to European patients as soon as possible.”

The marketing authorisation application is based on data from the Phase III HOPE atudy 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.

The analysis of the complete data from the HOPE study further demonstrated that Oxbryta, at a daily dose of 1,500mg, resulted in durable improvements in haemoglobin levels and markers of haemolysis over 72 weeks of treatment. 

Oxbryta is approved in the US for the treatment of SCD in adults and children 12 years of age and older.