Posted by Michael Wonder on 04 Jun 2019
bluebird bio announces EU conditional marketing authorisation for Zynteglo (autologous CD34+ cells encoding βA-T87Q-globin gene) gene therapy for patients 12 years and older with transfusion-dependent β-thalassemia who do not have β0/β0 genotype
3 June 2019 - European marketing authorisation for Zynteglo follows the fastest assessment of an advanced therapy medicinal product as part of the European Medicines Agency’s Priority Medicines program.
bluebird bio announced today that the European Commission has granted conditional marketing authorization for Zynteglo (autologous CD34+ cells encoding βA-T87Q-globin gene), a gene therapy for patients 12 years and older with transfusion-dependent β-thalassemia (TDT) who do not have a β0/β0 genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen (HLA)-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.
