Bluebird Secures FDA Nod For Genetic Blood Disease Candidate

The U.S. Food and Drug Administration (FDA) has approved Bluebird bio’s BLUE lead asset ZYNTEGLO (betibeglogene autotemcel) to treat the underlying genetic cause of beta‑thalassemia in adult and pediatric patients who require regular red blood cell (RBC) transfusions.

The regulatory approval is based on data readout from Phase 3 studies HGB-207 (Northstar-2) and HGB-212 (Northstar-3), and the long-term follow-up study LTF-303.

The approval of ZYNTEGLO is the culmination of nearly 10 years of clinical research of gene therapy in patients with transfusion-dependent beta-thalassemia.

Andrew Obenshain, Chief executive officer, commented: "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. 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.”

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

FDA reviewed ZYNTEGLO under Priority Review, and the Company received a Priority Review voucher upon approval. The lead asset was previously granted Orphan Drug designation and Breakthrough Therapy designation.

Price Action : bluebird shares are trading around 9 percent higher at $7.39 on Wednesday during after-hours session.