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FDA Panel Endorses Bluebird Bio's Eli-cel Gene Therapy For Rare Neurodegenerative Disease

An advisory committee to the U.S. Food and Drug Administration has unanimously endorsed bluebird bio Inc.'s (BLUE) eli-cel gene therapy for a rare neurodegenerative disease.

The FDA's Cellular, Tissue, and Gene Therapies Advisory Committee or CTGTAC voted 15-0 in favour of the company's elivaldogene autotemcel or eli-cel for the treatment of early active cerebral adrenoleukodystrophy or CALD in patients less than 18 years of age who do not have an available and willing human leukocyte antigen or HLA-matched sibling hematopoietic stem cell or HSC donor.

If approved, eli-cel will be the first and only gene therapy for the treatment of early active Cerebral adrenoleukodystrophy, a rare neurodegenerative disease that primarily affects young children and leads to irreversible loss of neurologic function and death. Nearly half of patients who do not receive treatment die within five years of symptom onset. Allogeneic hematopoietic stem cell transplant (allo-HSCT) is currently the only effective treatment option but is associated with serious potential complications and mortality that increase in patients without a matched sibling donor.

The committee's recommendation was based on the Biologics License Application currently under priority review by the FDA with a PDUFA goal date set for September 16, 2022. The biologics license application for eli-cel is supported by efficacy and safety data from the completed Phase 2/3 Starbeam study (ALD-102) (N=32).

The committee also discussed the overall safety of lentiviral vector gene therapies, concluding in a 13 to 1 vote that the safety data from lovo-cel for sickle cell disease is not relevant to the review of eli-cel.

In addition to granting eli-cel BLA priority review, the FDA previously granted eli-cel Orphan Drug status, Rare Pediatric Disease designation, and Breakthrough Therapy designation. bluebird bio is eligible to receive a priority review voucher upon potential approval of eli-cel.

Earlier today, bluebird bio noted that the Nasdaq halted trading of the company's common stock on June 9 and June 10, 2022.

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