Skysona has become the first and only gene therapy approved in the EU to treat early Cerebral Adrenoleukodystrophy (CALD), a rare neurodegenerative disease that can lead to progressive, irreversible loss of neurologic function, and death.
The European Commission (EC) granted marketing authorisation for Skysona (elivaldogene autotemcel, Lenti-D) to bluebird bio for the treatment of early 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.
Previously, the only therapeutic option available to CALD patients was transplantation of stem cells from a donor, called allogeneic hematopoietic stem cell transplant (allo-HSCT), which is associated with severe potential complications and mortality that increase in patients without a matched sibling donor (MSD). It is estimated that more than 80% of patients diagnosed with CALD do not have an MSD.
Skysona was reviewed as part of the European Medicines Agency’s Priority Medicines scheme (PRIME) and was previously granted Orphan Medicinal Product status. The marketing authorization is valid in all 27 member states of the EU, as well as Norway, Liechtenstein, and Iceland.