Bluebird Bio Files US Application For β-thalassemia Gene Therapy

Bluebird Bio Inc BLUE completed the rolling submission of its marketing application to the FDA seeking approval for betibeglogene autotemcel (beti-cel) gene therapy.
The application covers adult, adolescent, and pediatric patients with β-thalassemia who require regular red blood cell (RBC) transfusions across all genotypes.
Read Next: Bluebird Bio's Shares Drop To 52-Week Low After Another Gene Therapy Trial Placed On FDA Hold.
The FDA previously granted beti-cel Orphan Drug status and Breakthrough Therapy designation for transfusion-dependent β-thalassemia (TDT).
If approved, beti-cel will be the first hematopoietic (blood) stem cell (HSC) ex-vivo gene therapy for patients in the U.S.
The submission is based on data from Phase 3 HGB-207 (Northstar-2) and HGB-212 (Northstar-3) studies and the Phase 1/2 HGB-204 (Northstar) and HGB-205 studies.
Together, these studies represent more than 220 patient-years of experience with beti-cel.
The data include two patients with up to seven years of follow-up, eight with at least six years of follow-up, and 19 with at least five years of follow-up.
Related content: Benzinga's Full FDA Calendar.
Price Action: BLUE stock gained 5.61% at $19 in premarket trading on the last check Wednesday.