- The FDA has accepted Bluebird bio Inc's BLUE marketing application for betibeglogene autotemcel (beti-cel) for β-thalassemia.
- The agency has granted priority review for Beti-cel, gene therapy for β-thalassemia across all genotypes who require regular red blood cell transfusions.
- If approved, beti-cel will be the first one-time treatment that addresses the underlying genetic cause of disease—offering an alternative to regular RBC transfusions and iron chelation therapy.
- The agency has set a Prescription Drug User Fee Act goal date of May 20, 2022.
- The application is based on data from Phase 3 studies HGB-207 (Northstar-2) and HGB-212 (Northstar-3), the Phase 1/2 HGB-204 (Northstar) and HGB-205 studies, and the long-term follow-up study LTF-303.
- Together, these studies represent more than 220 patient-years of experience with beti-cel.
- The FDA previously granted beti-cel Orphan Drug status and Breakthrough Therapy designation.
- Also See: Bluebird Bio To Withdraw Marketing Of Skysona Gene Therapy In Europe.
- Price Action: BLUE shares are up 10.30% at $11.75 during the premarket session on the last check Monday.
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