BLUE - FDA accepts for priority review bluebird bio's eli-cel gene therapy application

bluebird bio (NASDAQ:BLUE) announces that the FDA has accepted for priority review the Biologics License Application (BLA) for elivaldogene autotemcel (eli-cel, Lenti-D), a gene therapy for cerebral adrenoleukodystrophy (CALD) in patients less than 18 years of age. The agency set a PDUFA goal date of June 17, 2022. The BLA is supported by efficacy and safety data from the completed Phase 2/3 Starbeam study (ALD-102). Additionally, the BLA contains data for 23 subjects dosed in the Phase 3 ALD-104 study. CALD is a progressive and irreversible neurodegenerative disease that involves the breakdown of myelin, the protective sheath that nerve cells need to function effectively, especially for thinking and muscle control. On August 9, FDA palced clinical hold on eli-cel clinical program, following a suspected unexpected serious adverse reaction of myelodysplastic syndrome.

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FDA accepts for priority review bluebird bio's eli-cel gene therapy application