Cambridge, MA-based Bluebird said on June 3rd that the European Commission’s regulatory nod for its therapy, Zynteglo, is a conditional approval, meaning the decision was made with less data than typically required for new drugs. Such approvals are reserved for treatments addressing serious or life-threatening diseases, and Bluebird will be required to submit additional data as a part of an annual renewal process. The regulatory decision comes two months after a European Commission advisory committee recommended approval. Beta thalassemia is caused by a defect in the gene that codes for hemoglobin, the protein in red blood cells that carries oxygen. The disease’s severity depends on the amount of hemoglobin a patient’s body is able to produce. The lack of oxygen-rich blood throughout the body can lead patients to feel fatigue, shortness of breath, and headaches. In the most serious cases, patients require lifelong blood transfusions every two to four weeks. These transfusions can lead to a dangerous buildup of iron in organs that requires chelation therapy to remove it. Bone marrow transplants offer another treatment option, but not all patients qualify and, for those who do, the procedure comes with its own risks.
For further information, see Xconomy (https://xconomy.com/boston/2019/06/03/bluebird-bios-gene-therapy-for-blood-disease-wins-european-approval/)