Work

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

Year: 2026

Type: article

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.

Source: Orphanet Journal of Rare Diseases

DOI: 10.1186/s13023-026-04585-y

Authors: Francesca Severino, Stefano Benvenuti, Giuliana Ferrari, Alessandro Aiuti

Institutions: Vita-Salute San Raffaele University, Fondazione Telethon, IRCCS Ospedale San Raffaele, The San Raffaele Telethon Institute for Gene Therapy

Language: English

Topic: Pharmaceutical Economics and Policy

Subfield: Economics and Econometrics

Field: Economics, Econometrics and Finance

Domain: Social Sciences

SDG: Good health and well-being

Open Access status: gold

Estimated APC paid: $2,990