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 dise…