bluebird bio Receives Positive CHMP Opinion for SKYSONA™ (elivaldogene autotemcel, Lenti-D™) Gene Therapy for Patients Less Than 18 Years of Age with Early Cerebral Adrenoleukodystrophy (CALD)
bluebird bio, Inc. (Nasdaq: BLUE) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending marketing authorization for 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 stem cell (HSC) donor is not available. If approved by the European Commission (EC), SKYSONA will be the first one-time gene therapy approved to treat CALD, a rare neurodegenerative disease that occurs in childhood and can lead to progressive, irreversible loss of neurological function and death.
The CHMP’s positive opinion will now be reviewed by the EC, which has the authority to grant marketing authorization for SKYSONA in the European Union (EU). A CHMP positive opinion is one of the final steps before the EC decides whether to authorize a new medicine. A final decision by the EC for SKYSONA is anticipated in mid-2021. SKYSONA is not approved for any indication in any geography.
“The goal of treatment with SKYSONA is to stabilize disease progression in children with CALD for whom a matched sibling donor is not available, in order to prevent further neurological decline and improve survival for these young patients,” said Richard Colvin, M.D., Ph.D., interim chief medical officer, bluebird bio. “This positive opinion from the CHMP marks the first regulatory approval recommendation for any gene therapy for CALD, bringing us closer to a one-time, durable treatment option that stabilizes neurological disease while reducing the risk of the serious immune complications associated with allogeneic stem cell transplantation (allo-HSCT), which is the only therapeutic option for children with this devastating disease. Together with the ALD community and clinical investigators, we are all optimistic that new hope could soon be provided to patients suffering from this unbearable condition.”
Adrenoleukodystrophy (ALD) is a rare, X-linked metabolic disorder that primarily affects males; worldwide, an estimated one in 21,000 male newborns are diagnosed with ALD. The disorder is caused by mutations in the ABCD1 gene that affect the production of adrenoleukodystrophy protein (ALDP) and subsequently cause toxic accumulation of very long-chain fatty acids (VLCFAs), primarily in the adrenal gland and white matter of the brain and spinal cord. Approximately
“The rapid and progressive decline of cognitive and physical functions for a child living with CALD is agonizing for parents, families and healthcare providers to witness. With CALD, each day truly counts, as nearly half of the patients who do not receive treatment will die within five years of symp
