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Bluebird’s Beta-Thalassemia Gene Therapy Faces US Panel Scrutiny On Hematologic Malignancy Risk

 
• By Sue Sutter

Although there were no cases of hematological malignancy reported and no evidence of insertional oncogenesis in patients treated with beti-cel, FDA raises a theoretical risk due to delayed platelet engraftment, as well as malignancies seen in other lentiviral vector programs.

Fireworks
Beti-cel administration led to transfusion independence for the majority of TDT patients in Phase III trials. • Source: Shutterstock

A US Food and Drug Administration advisory committee will consider recommendations for pretreatment screening and postmarketing safety monitoring to address the potential risk of hematologic malignancies with bluebird bio’s betibeglogene autotemcel (beti-cel), a lentiviral vector-based gene therapy for beta-thalassemia.

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