BLUE - EMA accepts Bluebird bio's marketing application for eli-cel gene therapy for neurodegenerative disorder

The European Medicines Agency ((EMA)) has accepted Bluebird bio's (BLUE) marketing authorization application for its investigational elivaldogene autotemcel (eli-cel, Lenti-D) gene therapy for the treatment of patients with cerebral adrenoleukodystrophy ((CALD)), a fatal neurodegenerative disease primarily affecting young boys.In July 2020, EMA's advisory group granted an accelerated assessment to eli-cel, potentially reducing the review time of from 210 days to 150 days. The FDA granted eli-cel Orphan Drug status, Rare Pediatric Disease and Breakthrough Therapy designation for the treatment of CALD. Bluebird bio is currently on track to submit the BLA in mid-2021.

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EMA accepts Bluebird bio's marketing application for eli-cel gene therapy for neurodegenerative disorder