Bluebird Bio announces EU conditional marketing authorization for ZYNTEGLO
bluebird bio announced that the European Commission has granted conditional marketing authorization for ZYNTEGLO, a gene therapy for patients 12 years and older with transfusion-dependent beta-thalassemia who do not have a beta0/beta0 genotype, for whom hematopoietic stem cell transplantation is appropriate but a human leukocyte antigen-matched related HSC donor is not available. bluebird bio will continue the country-by-country reimbursement process to help ensure access to ZYNTEGLO for appropriate patients. TDT is a severe genetic disease caused by mutations in the beta-globin gene that result in reduced or absent hemoglobin. In order to survive, people with TDT maintain hemoglobin levels through lifelong chronic blood transfusions. These transfusions carry the risk of progressive multi-organ damage due to unavoidable iron overload. ZYNTEGLO is a one-time gene therapy that addresses the underlying genetic cause of TDT and offers patients 12 years and older who do not have a beta0/beta0 genotype the potential to become transfusion independent, which once achieved is expected to be life-long. ZYNTEGLO was reviewed as part of the European Medicines Agency's Priority Medicines and Adaptive Pathways programs, which support medicines that may offer a major therapeutic advantage over existing treatments, or benefit patients without treatment options.