The U.S. Food and Drug Administration's (FDA) Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) unanimously voted in favour of Bluebird bio’s (NASDAQ:BLUE) elivaldogene autotemcel (eli-cel) for the treatment of early active cerebral adrenoleukodystrophy (CALD) in patients less than 18 years of age.
The advisory committee's recommendation is based on the Biologics License Application (BLA) granted priority review by the FDA with a PDUFA goal date set for September 16, 2022.
The BLA for elivaldogene autotemcel is supported by efficacy and safety data from the completed Phase 2/3 Starbeam study.
The Cerebral adrenoleukodystrophy (CALD) is a rare neurodegenerative disease that primarily affects young children and leads to irreversible loss of neurologic function and death.
Andrew Obenshain, chief executive officer, commented : “For decades, the CALD community has fought for the opportunity to stave off the rapid, irreversible decline associated with this devastating disease. Today we are one step closer to delivering a potentially lifesaving therapy for CALD. We are grateful to the families, clinicians and committee members who participated in today's advisory committee discussion and remain committed to working with the FDA as it completes its review of the eli-cel Biologics License Application”.
The lead drug, eli-cel was granted Orphan Drug status, Rare Pediatric Disease designation, and Breakthrough Therapy designation by the regulatory agency.
The FDA considers the recommendations of Advisory Committee when making decisions on Biologics License Application (BLA).
If approved, elivaldogene autotemcel will be the first and only gene therapy for the treatment of early active CALD.
Nasdaq has halted trading of the Bluebird’s common stock on Thursday, June 9 and Friday, June 10, 2022.