[7][8] Elivaldogene autotemcel is indicated for the treatment of people with early, active CALD in boys aged 4 to 17 for whom a matched hematopoietic stem cell donor is not available.
[14] Cerebral adrenoleukodystrophy is caused by a mutation in the ABCD1 gene on the X chromosome, which codes for the ALD protein that helps transport very long chain fatty acids (VLCFAs) to peroxisomes for degradation.
[15] Patients with a dysfunctional ABCD1 gene lack a functional ALD protein, causing VLCFAs to improperly degrade and abnormally accumulate in the blood and central nervous system.
[18] Elivaldogene autotemcel has a black box warning for hematological cancers, as patients have developed myelodysplastic syndrome (MDS) due to lentiviral integration into proto-oncogenes.
[26][6] The applicant was Bluebird Bio (Netherlands) B.V.[6] In July 2021, the European Commission approved elivaldogene autotemcel under the tradename Skysona for CALD patients who have certain genetic mutations and don't have a sibling who is a match for a stem cell transplant.
[27] In July 2021, after receiving marketing authorization through the EMA, bluebird bio reported it planned to close operations and clinical trials in Europe, citing an inability to come to an agreement regarding reimbursement for therapy cost.
[28] This decision came after the withdrawal of Zynteglo, a gene therapy for severe beta thalassemia, from Germany in 2021 due to similar difficulties in reaching reimbursement agreements.