General MedicinePrescription OnlyPregnancy Cat. Not classifiedFDA Yes
Skysona
ELIVALDOGENE AUTOTEMCEL
1 INDICATIONS AND USAGE 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) an...
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Brand names:Skysona
Quick Reference
Medicine ClassGeneral Medicine
FDA ApprovedYes
PregnancyCategory Not classified
Quick Facts
- Active ingredient
- ELIVALDOGENE AUTOTEMCEL
- Medicine class
- General Medicine
- Brand names
- Skysona
- Availability
- Prescription only (Rx)
- Pregnancy category
- Not classified
- FDA approved
- Yes
What Is Skysona?
1 INDICATIONS AND USAGE 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) an...
Skysona (ELIVALDOGENE AUTOTEMCEL) belongs to the General Medicine class of medications. It was first approved by the FDA in Yes. This medication requires a prescription from a licensed healthcare provider.
Key Safety Information
- •Always follow the dose recommended by your healthcare professional. Taking more than prescribed increases the risk of side effects including serious complications.
- •Seek emergency care if you experience signs of an allergic reaction — hives, swelling, difficulty breathing, or rash. Stop the medication and call your doctor immediately.
- •Some medications can increase the risk of cardiovascular events. Tell your doctor if you have high blood pressure, a history of heart attack or stroke, or any pre-existing health condition.
- •Avoid or limit alcohol while taking Skysona. Do not drink alcohol without first discussing it with your healthcare provider as it may increase adverse effects.
- •Take Skysona with food or a full glass of water if stomach upset occurs. Stop use and talk to your doctor if you experience persistent stomach pain or GI symptoms.
- •Tell your doctor about all medications, supplements, and vitamins you take. Some combinations significantly increase the risk of harm.
This is a summary only. Always read the full prescribing information and consult your healthcare provider for personalized medical advice.
Uses & Indications
Skysona is prescribed for the following conditions. Some uses are FDA-approved indications; others may be evidence-based off-label uses. Consult your healthcare provider for personalized guidance.
1 INDICATIONS AND USAGE 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-
This indication is approved under accelerated approval based on 24-month Major Functional Disability (MFD)-free survival [see Clinical Studies (14) ] .
Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).
SKYSONA is an autologous hematopoietic stem cell-based gene therapy 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 CALD 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-
This indication is approved under accelerated approval based on 24-month Major Functional Disability (MFD)-free survival.
[see Clinical Studies (14) ] .
Dosage Guide
The following are general dosing guidelines only. Your actual dose should be determined by your healthcare provider based on your condition, renal/hepatic function, and other medications.
AD
Adult Dosage
2 DOSAGE AND ADMINISTRATION 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+ cel
Available Forms
Available Strengths
Side Effects
Common
- 6 ADVERSE REACTIONS 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;
Serious
- 5 WARNINGS AND PRECAUTIONS 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.
Rare
Medicine Interactions
Always inform your healthcare provider and pharmacist about ALL medications you take, including prescriptions, OTC medicines, vitamins, and supplements.
MajorModerateMinor
Warnings & Contraindications
5 WARNINGS AND PRECAUTIONS 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 engr
How Skysona Works
12.1 Mechanism of Action 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. Functional ALDP can then participate in the local degradation of very long chain fatty acids (VLCFAs), which is believed to slow or possibly prevent further inflammation and demyelination.
Pharmacokinetics
Absorption
absorption, distribution, metabolism, and elimination are not applicable
Metabolism
metabolism, and elimination are not applicable
Pregnancy & Breastfeeding Safety
Not classified
Breastfeeding
Many medications pass into breast milk in varying amounts. Before using Skysonawhile breastfeeding, discuss the benefits and risks with your healthcare provider or pharmacist — they can weigh your dose, your infant's age, and available lactation safety data to find the safest option for you and your baby.
Storage & Handling
Match the identity of the patient with the patient identifiers on the metal cassette(s), infusion bag(s), and Lot Information Sheet upon receipt. Store SKYSONA frozen in the vapor phase of liquid nitrogen at less than or equal to -140°C (-220°F) until ready for thaw and administration. Thaw SKYSONA
Frequently Asked Questions
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Skysona side effects
Complete adverse effect profile including common, serious, and rare reactions
Sources & References
- • FDA Prescribing Information — Official labeling and package insert for Skysona
- • U.S. National Library of Medicine — MedlinePlus drug information
- • American Hospital Formulary Service (AHFS) — Clinical pharmacology monograph
- • PubMed / MEDLINE — View clinical studies
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Medical Disclaimer
The information on this page is for educational purposes only and is not intended as medical advice, diagnosis, or treatment. Always consult your doctor, pharmacist, or qualified healthcare provider before starting, stopping, or changing any medication.