Mon. 22 Nov 2021, 6:54am ETBenzinga
In: Biotech, News, Health Care, Small Cap, FDA, Movers, Trading Ideas, General
  • The FDA has accepted Bluebird bio Inc's (NASDAQ: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.