Posted by Michael Wonder on 22 Jul 2021
bluebird bio receives EC approval for Skysona (elivaldogene autotemcel, Lenti-D) gene therapy for patients less than 18 years of age with early cerebral adrenoleukodystrophy without matched sibling donor
21 July 2021 - Skysona is the first and only gene therapy approved in the European Union to treat early CALD.
bluebird bio today announced that the European Commission has granted marketing authorisation of Skysona (elivaldogene autotemcel, Lenti-D), a one-time gene therapy for the treatment of early cerebral adrenoleukodystrophy in patients less than 18 years of age with an ABCD1 genetic mutation, and for whom a human leukocyte antigen matched sibling haematopoietic stem cell donor is not available.
