Elivaldogene Autotemcel
Elivaldogene Autotemcel (SKYSONA) is an autologous gene therapy approved for the treatment of cerebral adrenoleukodystrophy (CALD) in patients with ABCD1 mutations. The therapy involves collecting a patient's own CD34+ stem cells and genetically modifying them with a lentiviral vector to add functional copies of the ABCD1 gene before infusing them back into the patient. It is specifically indicated for patients with CALD who do not have a matched sibling donor available for hematopoietic stem cell transplantation.
SKYSONA is a one-time gene therapy for cerebral adrenoleukodystrophy that modifies a patient's own stem cells to slow or prevent neurological deterioration.
This medication carries a boxed (black box) warning — the strongest type of FDA warning. Review the full prescribing information before use.
Key facts at a glance
- ATC
- A16AX21
- RxNorm
- 2612527
- Generic
- Elivaldogene Autotemcel
- Class
- atc-a16
- Brands
- Skysona
- Availability
- rx
- Lactation
- unknown
- Routes
- —
- Forms
- injection
- Regions
- US, EU, CN, JP
- Monitoring
- —
This page is for informational purposes only.
Do not use this information to diagnose yourself or to replace a physician's advice. Always consult a qualified healthcare provider before starting, stopping, or changing any medication. If you believe you are experiencing a medical emergency, call your local emergency number immediately.
Dosage and administration
- Usual dose
- 1.8 x 10^6 CD34+ cells/kg
- Maximum
- consult physician
- Frequency
- single infusion
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 2.0 x 10^6 CD34+ cells/kg
- Maximum
- consult physician
- Frequency
- single infusion
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 1.8 x 10^6 CD34+ cells/kg
- Maximum
- consult physician
- Frequency
- single infusion
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
Overdose information
Special Populations
8 groupspregnancy
first trimesteravoid if possible
avoid use during pregnancy unless benefits significantly outweigh risks; women of childbearing potential should use effective contraception
regular fetal monitoring and ultrasound assessments
Use of this medication in this population requires careful consideration of risks and benefits.
pregnancy
second trimesteravoid if possible
avoid use during pregnancy unless benefits significantly outweigh risks
regular fetal monitoring including growth assessments
Use of this medication in this population requires careful consideration of risks and benefits.
pregnancy
third trimesteravoid if possible
avoid use during pregnancy; consider delaying treatment until after delivery if clinically appropriate
fetal monitoring and assessment of fetal wellbeing
Use of this medication in this population requires careful consideration of risks and benefits.
lactation
breastfeedingunknown
it is unknown whether elivaldogene autotemcel is excreted in human breast milk; breastfeeding is not recommended during treatment and for a period after administration
no specific monitoring requirements established
pediatric_age
children 4 to 17 yearsno adjustment needed for indicated age group
indicated for patients 4 to 17 years of age with early cerebral adrenoleukodrophrophy; administered as single intravenous infusion following myeloablative conditioning
regular monitoring for engraftment success, vector copy number, and potential insertional mutagenesis; long-term follow-up required
geriatric
elderly 65 years and olderuse with caution; no specific dosing recommendations established
not specifically studied in geriatric populations; use with caution due to potential reduced organ function and comorbidities
comprehensive geriatric assessment and close monitoring of organ function
Use with caution and monitor for adverse effects.
renal_impairment
mild to moderate renal impairmentno specific dosage adjustment recommended based on current data
use with caution; limited clinical data in patients with renal impairment; assess renal function before and after treatment
monitor renal function including serum creatinine and estimated glomerular filtration rate
Use with caution and monitor for adverse effects.
hepatic_impairment
mild to moderate hepatic impairmentno specific dosage adjustment recommended based on current data
use with caution; limited clinical data in patients with hepatic impairment; assess liver function before treatment
monitor liver function tests including ALT, AST, bilirubin, and coagulation parameters
Use with caution and monitor for adverse effects.
Statistics and clinical data
Drug class
Cellular and gene therapy product classification
Indication
Therapeutic indication for early-onset MLD
FDA approval year
Regulatory approval timeline
Mechanism of action
Molecular mechanism of action
SKYSONA is indicated for the treatment of cerebral adrenoleukodystrophy (CALD), a rare and progressive neurological disorder caused by mutations in the ABCD1 gene, in patients age 3 to 17 years who have early-stage disease and lack a matched sibling donor for allogeneic hematopoietic stem cell transplant.
SKYSONA adds functional copies of the ABCD1 gene to the patient's own CD34+ hematopoietic stem cells through transduction with a lentiviral vector (Lenti-D). After infusion, these modified stem cells engraft in the bone marrow and give rise to cells that express functional adrenoleukodystrophy protein (ALDP), which helps break down very long-chain fatty acids (VLCFAs) that accumulate in CALD, thereby potentially slowing or halting disease progression.
SKYSONA is for autologous use only and must not be used if the patient tests positive for the presence of replication-competent lentivirus. Patients should be monitored for hematologic malignancies and insertional oncogenesis, which have been observed with similar gene therapies. Pre-treatment conditioning with busulfan is required before infusion. As a one-time therapy, the risks and benefits should be carefully considered versus allogeneic transplant when a matched sibling donor is available.
Contraindications
Conditions and patient states where this medication should be avoided or used with caution.
Do not use (1)
Known allergy to this medication or its components
Use caution (1)
Consult healthcare provider for benefits vs risks assessment
Official drug label
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This information is for educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment.
This content is for general informational and educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment. Always seek the guidance of a qualified healthcare provider with any questions you have about a medical condition, and never disregard professional medical advice or delay seeking it because of something you read here.
If you think you may have a medical emergency, call your local emergency number or go to the nearest emergency department immediately.
Medically reviewed by WellAlly Editorial Team Last reviewed July 26, 2026. · Read our editorial policy