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.
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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.
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.
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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.
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