Benzinga | Oct 2, 2020 05:26AM EDT

bluebird bio Announced EMAg Acceptance of Marketing Authorization Application for elivaldogene autotemcel (eli-cel, Lenti-D) Gene Therapy for Cerebral Adrenoleukodystrophy

bluebird bio, Inc. (NASDAQ:BLUE) today 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 2/3 Starbeam study (ALD-102) formed the basis of the MAA, which is also supported with data from the ongoing Phase 3 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.

Eli-cel is a one-time investigational gene therapy designed to add functional copies of the ABCD1 gene into a patient's own hematopoietic (blood) stem cells (HSCs) that have been transduced ex vivo with the Lenti-D lentiviral vector (LVV). The addition of a functional gene allows patients to produce the adrenoleukodystrophy protein (ALDP), which is thought to allow for the breakdown of very-long-chain fatty acids (VLCFAs) that accumulate to toxic levels in the brain. There is no need for donor HSCs from another person.

The EMA accepted eli-cel gene therapy for the treatment of CALD into its Priorities Medicines scheme (PRIME) in July 2018, and previously granted Orphan Medicinal Product designation to eli-cel. In July 2020, the Committee for Medicinal Products for Human Use (CHMP) of the EMA granted an accelerated assessment to eli-cel, potentially reducing the EMA's active review time of the MAA from 210 days to 150 days.

The U.S. Food and Drug Administration (FDA) granted eli-cel Orphan Drug status, Rare Pediatric Disease designation, and Breakthrough Therapy designation for the treatment of CALD. bluebird bio is currently on track to submit the Biologics License Application (BLA) in the U.S. in mid-2021.

Eli-cel is not approved for any indication in any geography.

bluebird bio is currently enrolling patients for a Phase 3 study (ALD-104) designed to assess the efficacy and safety of eli-cel after myeloablative conditioning using busulfan and fludarabine in patients with CALD. Contact clinicaltrials@bluebirdbio.com for more information and a list of study sites.

Additionally, bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-304) for patients who have been treated with eli-cel for CALD and completed two years of follow-up in bluebird bio-sponsored studies.

The Phase 2/3 Starbeam study (ALD-102) has completed enrollment