Gene Therapy for Beta Thalassemia Recommended for EU Approval

According to a story from Stockwatch, the gene therapy company bluebird bio, inc. recently announced that the European Medicine Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended that the company’s gene therapy ZYNTEGLO™ be given conditional marketing approval. This approval will be for the use of this medication as a treatment for transfusion-dependent beta thalassemia in patients that are 12 years or older. These patients must have a certain genotype and be eligible for stem cell transplant but not have a viable donor.

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.

A Potential Game Changer

The positive recommendation from CHMP is a critical step for the approval of ZYNTEGLO. Now, the verdict is in the hands of the European Commission, which has the power to officially grant authorization for marketing. The successful approval would herald a treatment breakthrough for beta thalassemia patients in the EU. This single use gene therapy has the potential to free these patients from a life time of blood transfusions and iron chelation therapy. 

The approval could result in dramatic improvements to quality of life for beta thalassemia patients. In a sense, the mechanism of ZYNTEGLO takes the place of a stem cell transplant procedure where instead of getting functional stem cells from a donor, a patient’s own stem cells are extracted and modified with a functional version of the mutated gene. Then, these cells are reintroduced into the patient’s body.


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