Regulators' Advice Can Make a Difference: European Medicines Agency Approval of Zynteglo for Beta Thalassemia

This commentary aims to demonstrate how frequent interactions between a medicines developer, regulators, Health Technology Assessment (HTA) bodies, and patients during the development can result in accelerated access by patients to an innovative product, in this case, a gene therapy medicinal product for beta-thalassemia. The majority of patients, who needed at least eight transfusions per year in the last two years before treatment, remain transfusion independent over 12 months after Zynteglo (bluebird bio (Netherlands) B.V., Utrecht, The Netherlands) administration. Early approvals, albeit based on robust data, are only possible if a well thought-through postapproval commitment plan is put in place.