EC Approves New Drug to Treat Hemolytic Anemia in Patients with Sickle Cell Disease

Global Blood Therapeutics, Inc. (GBT) announced on Feb. 16, 2022 that the European Commission (EC) has approved the use of Oxbryta (voxelotor) to treat hemolytic anemia caused by sickle cell disease (SCD) in patients age 12 years and older. According to the press release, voxelotor is the first medicine approved in Europe that directly inhibits the molecular basis of sickling and destruction of red blood cells (sickle hemoglobin polymerization) in SCD.

In SCD, red blood cells become rigid, sticky, and sickle shaped. This process causes red blood cells to break down (hemolysis). This destruction of red blood cells consequently causes low hemoglobin and low oxygen delivery in the body, or hemolytic anemia. Hemolysis combined with sickled cells causes pain and impedes the flow of blood and supply of oxygen throughout the body due to blockages in capillaries and small blood vessels. According to the press release, SCD affects approximately 52,000 people in Europe.

Voxelotor inhibits sickle hemoglobin polymerization, therefore also inhibiting hemolysis and hemolytic anemia. The medicine improves oxygen delivery throughout the body by increasing hemoglobin’s attraction to oxygen.

“Hemolytic anemia is the root cause of significant morbidity in people living with sickle cell disease, yet available treatment options are limited,” said Dr. Baba Inusa, consultant and professor of paediatric haematology, Guy’s and St. Thomas’ NHS Foundation Trust, London and chair of the National Haemoglobinopathy Panel in England, in the press release. “Treatment with Oxbryta has been shown to break the recurrent cycle of red blood cell sickling leading to their destruction—significantly improving patients’ hemoglobin levels and the blood’s oxygen-carrying capacity.”

Source: GBT