Bluebird’s Eli-Cel Gene Therapy Gets US Panel Nod For Some Cerebral Adrenoleukodystrophy Patients

 
• By Sue Sutter

Committee endorses use for those who lack a matched donor for allogeneic hematopoietic stem cell transplant and says the treatment should be an option for patients with a matched, but unrelated, donor.

Scale
The clinical benefit seen with eli-cel outweighs the risk of myelodysplastic syndrome, an FDA panel said. • Source: Shutterstock

More from US FDA Performance Tracker

More from Regulatory Trackers