Section 12 · Monograph
Elivaldogene autotemcel
Reviewed Nov 2026FDA label
SkysonaAutologous stem-cell gene therapy
- Forms
- IV suspension: autologous CD34+ gene-modified cells
- Dose
- Early active cerebral adrenoleukodystrophy, boys 4-17 (indication restricted in Aug 2025 to those without an available HLA-matched donor): single IV infusion after busulfan conditioning
- MOA
- Lentiviral ABCD1 gene transfer into CD34+ cells
Black Box Warning
- Hematologic malignancy: life-threatening myelodysplastic syndrome and acute myeloid leukemia have occurred; as of July 2025 hematologic malignancy had been diagnosed in 10 of 67 trial participants (15%); monitor with complete blood counts at least every 3 months and lifelong surveillance
Indications
- Early, active cerebral adrenoleukodystrophy (CALD) in boys 4 to 17 years of age; restricted (August 2025) to patients who do not have an available HLA-matched allogeneic hematopoietic stem cell donorFDA-labeled
Mechanism of Action
- Gene-modified autologous stem cells produce functional ALD protein (ALDP), aiming to halt progression of cerebral demyelination
Full Dosing
Adult / usual
- Single IV infusion of autologous CD34+ cells transduced with a lentiviral vector carrying ABCD1, after myeloablative busulfan conditioning
- Prefer HLA-matched allogeneic stem cell transplantation when a suitable donor exists
- Requires mobilization, apheresis and treatment at a qualified center
Contraindications
- None listed beyond hypersensitivity and conditioning-agent contraindications
Drug Interactions
- Busulfan conditioning interactions (including antiseizure drugs such as phenytoin that affect busulfan levels)
- Avoid live vaccines around conditioning
Adverse Effects
- Hematologic malignancy (MDS, AML)
- Cytopenias, delayed platelet engraftment, neutrophil engraftment failure
- Nausea, vomiting, stomatitis, alopecia
- Hypersensitivity
Monitoring Parameters
- Complete blood count at least every 3 months; bone marrow evaluation as indicated
- Integration-site/clonality analysis annually after the first year
- MRI (Loes score) and neurologic function; 15-year long-term follow-up
Pregnancy & Lactation
- Busulfan conditioning causes infertility and embryo-fetal toxicity; counsel on fertility preservation