Gene therapy for beta-thalassemia blood disorder one step closer to European approval

3-31-2019 thals patient
Child with beta-thalassemia. Image: Thalassemia and Child Welfare Group

The first gene therapy to treat a rare blood disorder is one step closer to approval [March 29] following a recommendation by European officials.

Lentiglobin, the gene therapy for beta-thalassemia developed by Cambridge, Mass.-based Bluebird Bio, was recommended for approval by the Committee for Medicinal Products for Human Use (CHMP), the drug-reviewing arm of the European Medicines Agency. A final approval decision is expected within the next three months.

Beta-thalassemia is a rare, inherited blood disease caused by a mutation in the beta-globin gene, which in turn, leads to the production of impaired red blood cells and severe anemia. Regular blood transfusions are the only effective treatment for beta-thalassemia patients today.

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Bluebird’s lentiglobin, administered once, uses an inactivated virus to insert a healthy, working copy of the beta-globin gene into patient’s bone marrow. Once implanted, healthy red blood cells are produced and the need for chronic blood transfusions is eliminated.

Related article:  GMO herbicide-tolerant canola poses little risk to human health, EU Food Safety Authority finds

Assuming approval, Bluebird’s next challenge will be convincing European countries to pay for a Zynteglo. The gene therapy is expected to carry a six-figure price tag, or just under, although exact pricing details won’t be disclosed until after approval is secured.

Read full, original post: First gene therapy to treat rare blood disease nears European approval

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