FDA Advisory Committee unanimously supports beti-cel gene therapy for people with beta-thalassemia who require regular red blood cell transfusions.- bluebird bio

“Despite advances in care, people living with the most severe form of beta-thalassemia still require frequent transfusions of healthy red blood cells to survive, tethering them to the healthcare system for life and increasing their risk for severe complications and early death,” said Andrew Obenshain, chief executive officer, bluebird bio. “Today’s advisory committee recommendation is recognition of the substantial body of clinical data that support beti-cel as a potentially curative treatment option for these patients. We are grateful to the members of the beta-thalassemia community who contributed to today’s discussion and remain committed to working with the FDA as it completes its review of the beti-cel Biologics License Application.”

The advisory committee’s recommendation is based on the Biologics License Application (BLA) currently under priority review by the FDA with a decision goal date set for August 19, 2022. The BLA is based on data from bluebird bio’s Phase III studies HGB-207 (Northstar-2) and HGB-212 (Northstar-3), the Phase 1/II HGB-204 (Northstar) and HGB-205 studies, and the long-term follow-up study LTF-303 as of March 2021. Additionally, as of the latest data cutoff date (August 2021), data from bluebird bio’s clinical development program represent 240 patient-years of experience with beti-cel and the longest available follow-up data in beta-thalassemia patients requiring regular RBC transfusions treated with a one-time gene therapy..