The Committee for Medicinal Products for Human Use (CHMP) at the European Medicines Agency (EMA) adopted a positive opinion recommending marketing authorization for Oxbryta® (voxelotor) for the treatment of haemolytic anaemia due to sickle cell disease (SCD) in patients 12 years of age and older as monotherapy or in combination with hydroxycarbamide.

Voxelotor is a small molecule which attaches and stabilises haemoglobin, preventing haemoglobin polymerization that causes sickling and destruction of red blood cells in SCD. This situation leads to episodes of vaso-occlusive crisis (VOC) and haemolytic anaemia.

Oxbryta would be the first medicine available in Europe that directly inhibits haemoglobin polymerization.

Currently, most patients with SCD are treated with hydroxycarbamide and crizanlizumab, medicines for preventing VOC. However, there is a high unmet need for medicines to treat haemolytic anaemia. Available treatment options are limited to blood transfusions and allogenic hematopoietic stem cell transplantation, but it requires a histocompatible donor. Therefore, new medicines for this manifestation of the disease are needed.

Oxbryta is currently approved in the United States for the treatment of SCD in adults and children 12 years of age and older.  In addition, the U.S. Food and Drug Administration (FDA) has agreed to expand the use of Oxbryta to children as young as 4 years old in December 2021.

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Stay tuned for further updates on the decision of the European Commission.