bluebird bio’s Zynteglo (betibeglogene autotemcel) Receives the US FDA’s Approval for the Treatment of Beta-Thalassemia


The US FDA has approved Zynteglo (ex-vivo LVV gene therapy) to treat beta‑thalassemia in adult & pediatric patients who required regular RBC transfusions
The approval was based on the P-III (Northstar-2 & 3) & the long-term follow-up (LTF-303) study to evaluate Zynteglo in 41 patients aged 4 to 34yrs. with beta‑thalassemia with non-β0/β0 & β0/β0 genotypes.  The results showed that 89% of patients achieved durable transfusion independence (TI) & remained transfusion-free for 12mos. along with normal or near normal total Hb levels
The company is also conducting a long-term follow-up (LTF-303) study to monitor the safety & effectiveness of LVV gene therapy for TDT patients who have participated in sponsored clinical studies for 15yrs. post-treatment

Ref: Businesswire | Image: bluebird bio