EMA accepts MAA for elivaldogene autotemcel (eli-cel, Lenti-D) Gene Therapy for cerebral adrenoleukodystrophy . bluebird bio Inc.,

bluebird bio, Inc. announced that the European Medicines Agency (EMA) accepted the company’s marketing authorization application (MAA) 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. “CALD is a devastating disease, often marked by rapid neurodegeneration, the development of major functional disabilities, and eventual death. The acceptance of the MAA for eli-cel is a critical milestone in our continued collaboration with the EMA to potentially deliver an autologous gene therapy for boys with CALD,” said Gary Fortin, Ph.D., SVP, severe genetic diseases, bluebird bio. “Data from clinical studies conducted in patients with early CALD suggest eli-cel stabilizes the progression of the disease. If approved, eli-cel would represent the first therapy for CALD that uses a patient’s own hematopoietic stem cells, potentially mitigating the risk of life-threatening immune complications associated with transplant using cells from a donor.” Data from the Phase II/III Starbeam study (ALD-102) formed the basis of the MAA, which is also supported with data from the ongoing Phase II ALD-104 study and the long-term follow-up study (LTF-304). The most recent results from these studies were presented at the 46th Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT 2020) in August 2020. bluebird bio is currently on track to submit the Biologics License Application (BLA) in the U.S. in mid-2021.