Beta-Thalassemia Gene Tx Gets Ringing Endorsement From FDA Advisers
An FDA advisory committee on Friday unanimously recommended betibeglogene autotemcel (beti-cel) for approval in patients with transfusion-dependent beta-thalassemia, a chronic inherited blood disorder. With a 13-0 vote, FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee said the benefits of the one-time, first-in-class lentiviral vector (LVV) gene therapy outweigh any of the risks associated with the […]