Global Blood Therapeutics Inc. (NASDAQ: GBT) has announced that the European Commission has given Oxbryta (voxelotor) Marketing Authorization to treat hemolytic anemia resulting from sickle cell disease in pediatric patients above 12 and adult patients as a monotherapy or a combination with hydroxycarbamide.
Oxybryta to prevent sickle hemoglobin polymerization
Oxbryta is a once-per-day oral therapy that would be the first drug in Europe to prevent sickle hemoglobin (HbS) synthesis directly, the structural foundation of sickling and red blood cell destruction in SCD.
Head of Europe and GCC at GBT, Sebastiana Stachowiak, said, “Living with sickle cell disease is a challenging life-long journey for patients and their families and can negatively impact every aspect of their lives. At GBT, our goal is to transform the treatment of sickle cell disease, and we are immensely proud to have developed an innovative medicine that we believe has the potential to change the trajectory of the disease.”
SCD is among Europe’s most common hereditary illnesses, affecting over 52,000 people, mainly of Mediterranean, African, and South Asian origin. Red blood cells in SCD lose their fluidity, becoming hard, sticky, and sickle-shaped or curved. The sickling process results in the breakdown of red blood cells (hemolysis), resulting in hemolytic anemia, which affects people with SCD to variable degrees. Hemolysis causes vasculopathy, which, in combination with sickled cells, causes obstructions in vessels and small blood arteries, producing discomfort and restricting the flow of blood and oxygen flow all through the body. Complications and comorbidities associated with SCD are gradual and life-threatening, including impairment to vital tissues such as the liver, lungs, kidneys, and heart.
Oxbryta approved in European Commission
The EC approval comes following a positive opinion from the Committee for Medicinal Products for Human Use in December 2021. Oxbryta now enjoys marketing permission in all EU countries, and also Iceland, Liechtenstein, and Norway, thanks to the EC clearance. In addition, GBT has applied to the Medicines and Healthcare Products Regulatory Agency (MHRA) in the United Kingdom for a British Marketing Authorisation under the EC Decision Reliance Procedure.
