bluebird bio Receives EC Approval for SKYSONA Gene Therapy

 One-time treatment with SKYSONA has been shown to have a durable effect in improving survival outcomes and preserving neurologic function across pivotal and long-term studies, with the longest follow-up of nearly seven years (82.7 months)

SKYSONA uses the patient’s own blood stem cells and there have been no reports of graft-versus-host disease (GVHD), graft failure or rejection, or transplant-related mortality (TRM) (n=51) across clinical studies to-date

bluebird bio, Inc. (Nasdaq: BLUE) today announced that the European Commission (EC) has granted marketing authorization of SKYSONA™ (elivaldogene autotemcel, Lenti-D™), a one-time gene therapy for the treatment of early cerebral adrenoleukodystrophy (CALD) in patients less than 18 years of age with an ABCD1 genetic mutation, and for whom a human leukocyte antigen (HLA)-matched sibling hematopoietic (blood) stem cell (HSC) donor is not available. SKYSONA is the first one-time gene therapy approved in the European Union (EU) to treat CALD, a rare neurodegenerative disease that occurs in childhood and can rapidly lead to progressive, irreversible loss of neurologic function, and death.

https://investor.bluebirdbio.com/news-releases/news-release-details/bluebird-bio-receives-ec-approval-skysonatm-elivaldogene