The withdrawal of beti-cel therapy for β-Thalassemia from the European market: a multistakeholder perspective

Abstract

Abstract This report aims to outline the complex and multifaceted framework within which the journey to European market access for a β-thalassemia gene therapy has unfolded, highlighting the clinical, ethical, economic, and regulatory challenges faced by all stakeholders involved. Focusing on betibeglogene autotemcel (Zynteglo), a gene therapy for β- thalassemia developed by the U.S. based biotech company Bluebird Bio, this paper analyzes official sources of information from the therapy developer, regulators, patients, health technology assessment bodies and payers with the aim of offering a measure of this complex operation that culminated in the therapy’s withdrawal from the European market in 2022.