Posted by Michael Wonder on 18 Dec 2021
bluebird bio announces FDA priority review of biologics license application for eli-cel gene therapy for cerebral adrenoleukodystrophy in patients without a matched sibling donor
17 December 2021 - If approved, eli-cel will be the first and only gene therapy for the treatment of cerebral adrenoleukodystrophy, a rare neurodegenerative disease primarily affecting young children that can lead to progressive, irreversible loss of neurologic function and death.
bluebird bio today announced that the U.S. FDA has accepted for priority review the biologics license application for elivaldogene autotemcel (eli-cel, Lenti-D), the company’s gene therapy for cerebral adrenoleukodystrophy in patients less than 18 years of age.
The agency set a Prescription Drug User Fee Act goal date of 17 June 2022.
