FDA announced that it had approved Zynteglo (betibeglogene autotemcel), a novel cell-based gene therapy for adult and pediatric patients with beta-thalassemia who need regular red blood cell transfusions.
Zynteglo, developed by Bluebird Bio (Nasdaq: BLUE), is administered as a single dose.
The product is indicated for adult and pediatric patients who need regular red blood cell transfusions.
Zynteglo represents the first ex-vivo lentiviral vector gene therapy to win FDA approval for beta-thalassemia.
To date, Zynteglo has been the subject of a dozen clinical trials, including for sickle cell anemia.
“Today’s approval is an important advance in the treatment of beta-thalassemia, particularly in individuals who require ongoing red blood cell transfusions,” said Dr. Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, in a news release. “Given the potential health complications associated with this serious disease, this action highlights the FDA’s continued commitment to supporting the development of innovative therapies for patients with limited treatment options.”
“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, Bluebird Bio CEO, in a news release. “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.”
Beta-thalassemia is a genetic blood disorder involving a low hemoglobin level and red blood cells.
Symptoms of the condition include dizziness, fatigue, shortness of breath and bone abnormalities.
A form of the disease known as transfusion-dependent beta-thalassemia often requires life-long red blood cell transfusions.