FDA approves first gene therapy for beta thalassemia

The U.S. Food and Drug Administration (FDA) has approved betibeglogene autotemcel (Zynteglo; bluebird bio) as a one-time gene therapy to treat the underlying genetic cause of beta‑thalassemia in patients who require regular red blood cell (RBC) transfusions, according to a press release.

“The FDA approval of Zynteglo offers people with beta-thalassemia the possibility of freedom from burdensome regular red blood cell transfusions and iron chelation, and unlocks new possibilities in their daily lives,” said Andrew Obenshain, chief executive officer, bluebird bio. “After more than a decade of research and clinical development, and through the perseverance of clinicians, patients, and their families, the approval of ZYNTEGLO marks a watershed moment for the field of gene therapy. As the first ex-vivo lentiviral vector gene therapy approved in the U.S. for the treatment of people with beta-thalassemia, we are ushering in a new era in which gene therapy has the potential to transform existing treatment paradigms for diseases that currently carry a lifelong burden of care.”

Due to the complex nature of gene therapy, Zynteglo will be available exclusively at Qualified Treatment Centers (QTCs) which are carefully selected based on their expertise in relevant areas such as stem cell transplantation, cell and gene therapy, and beta-thalassemia; and receive specialized training to administer Zynteglo.

Read the full press release here.