Elivaldogene Autotemcel

SKYSONA adds functional copies of the ABCD1 cDNA into patients' hematopoietic stem cells (HSCs) through transduction of autologous CD34+ cells with Lenti-D LVV. After SKYSONA infusion, transduced CD34+ HSCs engraft in the bone marrow and differentiate into various cell types, including monocytes (CD14 + ) capable of producing functional ALDP.

• SKYSONA is indicated to slow the progression of neurologic dysfunction in boys 4-17 years of age with early, active cerebral adrenoleukodystrophy (CALD) without an available human leukocyte antigen (HLA)-matched donor for allogeneic hematopoietic stem cell transplant. Early, active cerebral adrenoleukodystrophy refers to asymptomatic or mildly symptomatic (neurologic function score, NFS ≤ 1) boys who have gadolinium enhancement on brain magnetic resonance imaging (MRI) and Loes scores of 0.5-9.

• None. None.contraindicated

For autologous use only. For intravenous use only. For autologous use only. For intravenous use only. Patients must undergo hematopoietic stem cell (HSC) mobilization and apheresis to obtain CD34+ cells for SKYSONA manufacturing. ( 2.2 ) Dosing of SKYSONA is based on the number of CD34+ cells in the infusion bag(s) per kg of body weight. ( 2.1 ) The minimum recommended dose is 5.0 × 10 6 CD34+ cells/kg. ( 2.1 ) Full myeloablative and lymphodepleting conditioning must be administered before infusion of SKYSONA. ( 2.2 ) Verify the patient's identity matches the unique patient identification information on the SKYSONA infusion bag(s) prior to infusion. ( 2.2 ) Do not sample, alter, or irradiate SKYSONA. ( 2.2 ) Do not use an in-line blood filter or an infusion pump. ( 2.3 ) 2.1 Dose SKYSONA is provided as a single dose for infusion containing a suspension of CD34+ cells in one or two infusion bags. The minimum recommended dose of SKYSONA is 5.0 × 10 6 CD34+ cells/kg. The dose is calculated based on the patient's weight prior to first apheresis. See the Lot Information Sheet provided with the product shipment for additional information pertaining to dose. 2.2 Preparation Before SKYSONA Infusion Before mobilization, apheresis, and conditioning are initiated, confirm that hematopoietic stem cell (HSC) transplantation is appropriate for the patient.

Serious Infections: Life-threatening bacterial and viral infections may occur. Monitor patients for signs and symptoms of infection. ( 5.2 ) Prolonged Cytopenias: Patients may exhibit cytopenias >1 year after treatment. Monitor patients for bleeding and infection. ( 5.3 ) Delayed Platelet Engraftment: Monitor patients for thrombocytopenia and bleeding until platelet engraftment and count recovery. ( 5.4 ) Risk of Neutrophil Engraftment Failure: Monitor absolute neutrophil counts and if neutrophil engraftment does not occur, give rescue cells. ( 5.5 ) 5.1 Hematologic Malignancy Hematologic malignancies, including myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML), have developed in patients treated with SKYSONA in clinical studies between 14 months and 10 years after SKYSONA administration [see Adverse Reactions (6.1) ] . Malignancies are life-threatening. Death related to treatment for malignancy and relapse of malignancy have occurred . SKYSONA Lenti-D lentiviral vector genomic integration, including into the proto-oncogene MECOM , appears to have mediated the cases of hematologic malignancy. All patients treated with SKYSONA in clinical studies have integrations into MECOM ; however, it is unknown which integrations into MECOM or other genes are likely to lead to malignancy. Consider consultation with hematology experts prior to SKYSONA treatment to inform benefit-risk treatment decision and to ensure adequate monitoring for hematologic malignancy.

Most common non-laboratory adverse reactions (≥ 20%): mucositis, nausea, vomiting, febrile neutropenia, alopecia, decreased appetite, abdominal pain, constipation, pyrexia, diarrhea, headache, rash. ( 6.1 ) Most common Grade 3 or 4 laboratory abnormalities (≥ 40%): leukopenia, lymphopenia, thrombocytopenia, neutropenia, anemia, hypokalemia. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Genetix Biotherapeutics at 1-833-999-6378 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. The safety data described in this section reflect exposure to a single dose of SKYSONA in 67 patients with CALD. Data were obtained from two single-arm trials and, for 36 patients, from a long-term follow-up study [see Clinical Studies (14) ] . The median (min, max) age across studies was 6 (4, 14) years; 100% were male; 54% were White/Caucasian, 4% were Black or African American, 1% were Asian, 10% were of other races including mixed race, and 30% did not report race; 25% were of Hispanic ethnicity. The median (min, max) duration of follow-up at time of initial approval was 24 (1, 88) months. In the two trials, serious adverse reactions from Day 1 (SKYSONA infusion) to last follow-up occurred in 54% of patients.

8.1 Pregnancy Risk Summary There are no available data with SKYSONA administration in pregnant women. Consider the risks associated with mobilization and conditioning agents on pregnancy and fertility. No animal reproductive and developmental toxicity studies have been conducted to assess whether SKYSONA can cause fetal harm when administered to a pregnant woman. No nonclinical germline transmission studies have been conducted with SKYSONA. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively. 8.2 Lactation Risk Summary There is no information regarding the presence of SKYSONA in human milk, the effect on the breastfed infant, and the effects on milk production. 8.3 Females and Males of Reproductive Potential Contraception Consult the Prescribing Information of the mobilization and conditioning agents for information on the need for effective contraception. There are insufficient exposure data to provide a precise recommendation on duration of contraception following treatment with SKYSONA.

Metabolism

SKYSONA is an autologous gene therapy which includes HSCs that have been genetically modified ex vivo . The nature of SKYSONA is such that conventional studies on pharmacokinetics, absorption, distribution, metabolism, and elimination are not applicable.

1. 1Dysmegakaryopoiesis220%
2. 2Thrombocytopenia220%
3. 3Biopsy Bone Marrow Abnormal110%
4. 4Cough110%
5. 5Cytogenetic Abnormality110%
6. 6Disease Progression110%
7. 7Myelodysplastic Syndrome With Excess Blasts110%
8. 8Neutropenia110%
9. 9Pancytopenia110%
10. 10Pyrexia110%
11. 11Tachycardia110%
12. 12Wheezing110%

The 4 most recently updated of 4 ClinicalTrials.gov registrations naming Elivaldogene Autotemcel as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• A Study of Participants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel
  Recruiting · Observational · 120 enrolled · Genetix Biotherapeutics Inc.
  NCT06224413updated 2025-07-02
• Long-term Follow-up of Participants With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product
  Active not recruiting · Observational · 64 enrolled · Genetix Biotherapeutics Inc.
  NCT02698579updated 2025-02-20
• A Clinical Study to Assess the Efficacy and Safety of Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy (CALD)
  Completed · Phase 3 · Interventional · 35 enrolled · Genetix Biotherapeutics Inc.
  NCT03852498updated 2024-05-24
• A Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD)
  Completed · Phase 2 · Phase 3 · Interventional · 32 enrolled · Genetix Biotherapeutics Inc.
  NCT01896102updated 2022-04-25

Frequently asked questions

How does Elivaldogene Autotemcel work?

SKYSONA adds functional copies of the ABCD1 cDNA into patients' hematopoietic stem cells (HSCs) through transduction of autologous CD34+ cells with Lenti-D LVV. After SKYSONA infusion, transduced CD34+ HSCs engraft in the bone marrow and differentiate into various cell types, including monocytes (CD14 + ) capable of producing functional ALDP.

What is Elivaldogene Autotemcel used for?

According to FDA labeling, Elivaldogene Autotemcel carries indications including: SKYSONA is indicated to slow the progression of neurologic dysfunction in boys 4-17 years of age with early, active cerebral adrenoleukodystrophy (CALD) without an available human leukocyte antigen (HLA)-matched donor for allogeneic hematopoietic stem cell transplant. Early, active cerebral adrenoleukodystrophy refers to asymptomatic or mildly symptomatic (neurologic function score, NFS ≤ 1) boys who have gadolinium enhancement on brain magnetic resonance imaging (MRI) and Loes scores of 0.5-9.. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Elivaldogene Autotemcel?

Elivaldogene Autotemcel is classified as Various alimentary tract and metabolism products, Gene Transduction or Replacement.

What are the brand names for Elivaldogene Autotemcel?

Elivaldogene Autotemcel is marketed under brand names including Skysona.

What are the contraindications for Elivaldogene Autotemcel?

Elivaldogene Autotemcel labeling lists contraindications including: None. None.. Always consult the full prescribing information and a clinician.