Bluebird Bio’s Gene Therapy for Blood Disease Wins European Approval
Summary
Cambridge, MA-based Bluebird said Monday 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 European approval for Zynteglo is for patients 12 and older who depend on blood transfusions to treat the disorder and can’t find a donor for a stem cell transplant. In Phase 1/2 clinical trials, Bluebird reported that 11 of 14 patients treated were able to stop transfusion treatments, and maintained “transfusion independence” until the cutoff of the study, which was as long as 56 months. Conditional approval in Europe is for one year, but Bluebird can renew it annually as long as the additional data submitted continue to show that the treatment’s benefits outweigh the risks.
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