Elivaldogene autotemcel
gene therapy
Elivaldogene autotemcel, sold under the brand name Skysona, is a gene therapy used to treat cerebral adrenoleukodystrophy (CALD). It was developed by Bluebird Bio and was given breakthrough therapy designation by the US Food and Drug Administration in May 2018.
Elivaldogene autotemcel is made specifically for each recipient, using the recipient's hematopoietic stem cells.
It was approved for medical use by the U.S. Food and Drug Administration in September 2022.
Medical uses
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. Early, active CALD refers to asymptomatic or mildly symptomatic boys with gadolinium enhancement on brain MRI and a Loes score of 0.5-9, a scale that rates the severity of CALD white matter lesions on a scale of 0 (normal) to 34 (abnormal) in adrenoleukodystrophy.
Elivaldogene autotemcel is a form of autologous hematopoietic stem cell therapy where stem cells are mobilized and collected from the patient and genetically modified to carry a functional copy of the ABCD1 gene using a lentiviral vector. Patients undergo myeloablative chemotherapy conditioning to kill stem cells in the bone marrow before infusion with elivaldogene autotemcel, which allows their modified stem cells to replace stem cells lacking a functional copy of the ABCD1 gene.
Elivaldogene autotemcel is a one-time treatment given as an autologous intravenous infusion. Dose depends on the patient's body weight.
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