FDA approves bluebird neurodegenerative gene therapy

Sept. 21, 2022

Earlier this week, bluebird bio announced that the FDA had granted Accelerated Approval to its gene therapy eli-cel, to be sold as Skysona. Eli-cel is now approved for the treatment of Early, Active Cerebral Adrenoleukodystrophy (CALD), in boys and teenagers aged 4-17.

CALD is a progressive, neurodegenerative disease that primarily impacts young boys, and causes a cascade of disabilities ranging from cortical blindness, total incontinence, and complete loss of voluntary movement. Almost half of the patients die if they do not receive treatment within five years of the disease first showing signs. 

According to bluebird, eli-cel works by providing a treatment consisting of transduced hematopoietic stem cells into cerebral microglia expressing the adrenoleukodystrophy protein, (ALDP),  helping to produce functional protein levels in the brain. In total, the biopharma has treated 67 patients with up to 7 years follow-up. 

The recent press release also shared that bluebird anticipates that the drug will be on the market by the end of this year and will be sold in the U.S. for $3M.