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.

Related article:  For untreatable diseases, should you consider unapproved stem cell therapy?

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.

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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