FDA Pushes Review Period For Bluebird bio's Neurodegenerative Disease, Thalassemia Gene Therapies

The FDA has extended the review period for Bluebird bio Inc's BLUE biologics licensing applications (BLA) for its lentiviral vector gene therapies.

• The agency has pushed the applications for betibeglogene autotemcel (beti-cel) for β-thalassemia and elivaldogene autotemcel (eli-cel) for cerebral adrenoleukodystrophy (CALD). 
• The revised PDUFA goal dates for beti-cel and eli-cel are August 19, 2022, and September 16, 2022.
• Related: Bluebird bio's Neurodegenerative Disease Gene Therapy Under Priority FDA Review, Despite Clinical Hold.
• The FDA extended the PDUFA goal dates for beti-cel and eli-cel to allow time to review additional clinical information previously submitted by the Company in response to FDA information requests.
• The extension of the FDA review timeline does not relate to new safety events for either beti-cel or eli-cel.
• The FDA accepted bluebird's BLA submission for beti-cel for β-thalassemia for priority review in November 2021.
• Bluebird also said that it received written questions from the FDA on partial clinical hold for the lovotibeglogene autotemcel (lovo-cel) gene therapy program for sickle cell disease patients under 18 years.
• The Company continues to evaluate the impact of the partial clinical hold on its projected Q1 2023 timing for submitting the BLA. 
• Price Action: BLUE shares are down 2.63% at $8.50 during the premarket session on the last check Tuesday.