BLUE - EMA advisory group backs bluebird bio's Skysona in rare neurodegenerative disease

bluebird bio (BLUE) announces that the Committee for Medicinal Products for Human Use ((CHMP)) of the EMA has adopted 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.CALD is 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 European Commission ((EC)), with final decision anticipated in mid-2021.If approved, Skysona will be the first one-time gene therapy to treat CALD.

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EMA advisory group backs bluebird bio's Skysona in rare neurodegenerative disease