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FDA priority review of BLA for eli-cel gene therapy for cerebral adrenoleukodystrophy (CALD) in patients without a matched sibling donor.- bluebird bio.

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Last updated:20th Dec 2021
Published:19th Dec 2021
bluebird bio, Inc. has announced that the FDA has accepted for priority review the Biologics License Application (BLA) for elivaldogene autotemcel (eli-cel, Lenti-D ), the company’s gene therapy for cerebral adrenoleukodystrophy (CALD) in patients less than 18 years of age.

Eli-cel is an investigational one-time gene therapy, custom-designed to treat the underlying cause of this irreversible neurodegenerative disease and to stabilize neurologic function. The agency set a Prescription Drug User Fee Act (PDUFA) goal date of June 17, 2022.

If approved, eli-cel will be the first approved treatment to address the underlying genetic cause of disease for patients living with CALD in the U.S. – offering appropriate patients an alternative to allogeneic hematopoietic stem cell transplant (allo-HSCT), which is associated with serious potential complications and mortality that increase in patients without a matched sibling donor. It is estimated that more than 70% of patients diagnosed with CALD do not have a matched sibling donor.

Condition: Cerebral Adrenoleukodystrophy
Type: drug

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