According to a story from BioPortfolio, the gene therapy company bluebird bio, Inc. recently announced that its gene therapy ZYNTEGLO™ has earned conditional marketing authorization in the European Union from the European Commission. ZYNTEGLO is a gene therapy intended for patients aged twelve years or older with severe, transfusion dependent beta thalassemia. These patients must not carry the β0/β0 genotype. The approval of this gene therapy could dramatically change outcomes for patients that qualify to use it.
About Beta Thalassemia
Beta thalassemia is a group of genetic disorders which affect the blood. These disorders are characterized by the abnomal production of hemoglobin, the substance in red blood cells which allows them to transport oxygen. The severity of the disorder can vary widely from person to person and depends on the present mutation. These mutations affect the HBB gene which is found on chromosome 11. These mutations can be easily passed down within families. Symptoms of beta thalassemia major, the most severe form, include spleen problems, skeletal abnormalities, poor growth, anemia, liver problems, diabetes, osteoporosis, and heart failure. Treatment for beta thalassemia may include blood transfusions, surgery, and bone marrow transplant, which can be curative for some children. There is a need for improved treatments for patients with severe cases. To learn more about beta thalassemia, click here.
Why ZYNTEGLO is a Game Changer
ZYNTEGLO is designed as a single use gene therapy, meaning that it only has to be administered once for patients to experience long term (possibly lifelong) benefit. This therapy offers the potential for these beta thalassemia patients to become transfusion independent for the first time. Reliance on periodic blood transfusions carries the risk of severe complications as a result of iron overload.
The evaluation of the therapy was conducted through a program from the European Medicines Agency (EMA) called Priority Medicines and Adaptive Pathways, which allows for an expedited review process for drugs that show major advantages in comparison to current treatments.
In clinical trials, the majority of patients that used ZYNTEGLO were ultimately able to achieve and maintain transfusion independence. Clearly, this gene therapy has the potentially to revolutionize treatment for severe beta thalassemia.