bluebird bio gains second FDA AdCom boost, this time for beti-cel gene therapy

US biotech firm bluebird bio (Nasdaq: BLUE) on Friday received another booster to its prospects as it announced the positive outcome of the US Food and Drug Administration’s (FDA) Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) discussion of betibeglogene autotemcel (beti-cel) for the treatment of people with beta-thalassemia who require regular red blood cell (RBC) transfusions.

This adds to Thursday’s news that the CTGTAC had  unanimously endorsed eli-cel, an investigational LVV gene therapy for the treatment of cerebral adrenoleukodystrophy, in a 15 to 0 vote, which came as a surpise.

On the question “Do the benefits of beti-cel outweigh the risks for the treatment of subjects with transfusion-dependent beta-thalassemia?” the CTGTAC voted 13 (yes) to 0 (no).