bluebird bio gets EC approval for CALD gene therapy Skysona

Following a positive recommendation from the European Medicines Agency’s human medicines committee (CHMP) in May, the European Commission (EC) has granted marketing authorization of Skysona (elivaldogene autotemcel, Lenti-D), developed by US biotech bluebird bio (Nasdaq: BLUE), whose shares were up almost 4% at $27.33 in pre-market trading today.

Skysona is a one-time gene therapy that has been cleared 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.

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 causes toxic accumulation of very long-chain fatty acids (VLCFAs), primarily in the adrenal gland and white matter of the brain and spinal cord.