Posted by Michael Wonder on 04 Oct 2020
bluebird bio announces European Medicines Agency’s acceptance of marketing authorisation application for elivaldogene autotemcel (eli-cel, Lenti-D) gene therapy for cerebral adrenoleukodystrophy
2 October 2020 - European Medicines Agency will evaluate eli-cel application under accelerated assessment.
bluebird bio today announced that the EMA accepted the company’s marketing authorisation application for its investigational elivaldogene autotemcel (eli-cel, Lenti-D™) gene therapy for the treatment of patients with cerebral adrenoleukodystrophy (CALD). CALD is a fatal neurodegenerative disease primarily affecting young boys.
Data from the Phase 2/3 Starbeam study (ALD-102) formed the basis of the application, which is also supported with data from the ongoing Phase 3 ALD-104 study and the long-term follow-up study (LTF-304).
