Skysona

Skysona is an FDA-registered drug product whose active ingredient is ELIVALDOGENE AUTOTEMCEL. It is labelled by Genetix Biotherapeutics Inc. taken by intravenous and marketed as a cellular therapy product. Identifiers on file: NDC 73554-2111, application BLA125755, label effective 20260721. This page reproduces the FDA-approved label text so you can check what a product is, who makes it and how it is supplied before discussing it with a pharmacist or prescriber.

Across FDA labelling, products containing ELIVALDOGENE AUTOTEMCEL are most often described in connection with: early active, cerebral adrenoleukodystrophy, hematopoietic stem, indicated slow, progression neurologic, dysfunction boys, years age, cald without. Wording differs between labels, and the full indications section on this page takes precedence when it is present.

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 0.5-9. 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 0.5-9. 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). ( 1 ) Limitations of Use SKYSONA does not treat or prevent adrenal insufficiency. ( 1 ) An immune response to SKYSONA may cause rapid loss of efficacy of SKYSONA in patients with full deletions of the human adenosine triphosphate binding cassette, sub family D, member 1 ( ABCD1) gene. ( 1 ) SKYSONA has not been studied in CALD secondary to head trauma. ( 1 ) Given the risk of hematologic malignancy with SKYSONA, and unclear long-term durability of SKYSONA and human adrenoleukodystrophy protein (ALDP) expression, careful consideration should be given to the timing of treatment for each boy and treatment of boys with isolated pyramidal tract disease as clinical manifestations do not usually occur until adulthood. ( 1 ) Limitations of Use SKYSONA does not prevent the development of or treat adrenal insufficiency due to adrenoleukodystrophy. An immune response to SKYSONA may limit the persistence of descendent cells of SKYSONA, causing rapid loss of efficacy of SKYSONA in patients with full deletions of the human adenosine triphosphate binding cassette, sub family D, member 1 ( ABCD1) gene. SKYSONA has not been studied in patients with CALD secondary to head trauma. Given the risk of hematologic malignancy with SKYSONA, and unclear long-term durability of SKYSONA and human adrenoleukodystrophy protein (ALDP) expression, careful consideration should be given to the appropriateness and timing of treatment for each boy, especially for boys with isolated pyramidal tract disease since their clinical symptoms do not usually occur until adulthood.

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+ 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. Perform screening for hepatitis B virus (HBV), hepatitis C virus (HCV), human immunodeficiency virus 1 & 2 (HIV-1/HIV-2) and Human T-lymphotropic virus 1 & 2 (HTLV-1/HTLV-2) in accordance with clinical guidelines before collection of cells for manufacturing. Mobilization and Apheresis Patients are required to undergo HSC mobilization followed by apheresis to obtain CD34+ cells for product manufacturing. Weigh the patient prior to the first apheresis collection. Collect a minimum number of CD34+ cells of 12 × 10 6 CD34+ cells/kg. A back-up collection of CD34+ cells of ≥ 1.5 × 10 6 CD34+ cells/kg (if collected by apheresis) or ≥ 1.0 × 10 8 TNC/kg (Total Nucleated Cells, if collected by bone marrow harvest) is required. Collect and cryopreserve these cells prior to initiating conditioning and infusion with SKYSONA. The back-up collection may be needed for rescue treatment if there is: 1) compromise of SKYSONA after initiation of conditioning and before SKYSONA infusion, 2) primary engraftment failure, or 3) loss of engraftment after infusion with SKYSONA. Myeloablative and Lymphodepleting Conditioning Full myeloablative and lymphodepleting conditioning must be administered before infusion of SKYSONA. Consult prescribing information for the conditioning agents prior to treatment. Do not begin conditioning until SKYSONA has been received and stored at the treatment center and the availability of the back-up collection of CD34+ cells is confirmed. After completion of conditioning, allow a minimum of 48 hours of washout before SKYSONA infusion. Receipt and Storage of SKYSONA Ensure the availability of vapor phase of liquid nitrogen storage at less than or equal to -140°C (-220°F) at the treatment center. SKYSONA is shipped to the treatment center in the vapor phase of liquid nitrogen shipper. Confirm Patient Identifiers on the product labels and Lot Information Sheet within the shipper. If there are any concerns about the product or packaging upon receipt, contact Genetix Biotherapeutics at 1-833-999-6378. Keep the infusion bag(s) in the metal cassette(s) and transfer SKYSONA from the vapor phase of liquid nitrogen shipper to the treatment center vapor phase of liquid nitrogen storage at less than or equal to -140°C (-220°F) until ready for thaw and administration. Preparation of SKYSONA for Infusion Coordinate the timing of SKYSONA thaw and infusion. Confirm the infusion time in advance and adjust the start time of SKYSONA thaw such that it will be available for infusion when the patient and healthcare providers are ready. SKYSONA contains human blood cells that are genetically modified with replication-incompetent, self-inactivating Lenti-D lentiviral vector (LVV). Follow universal precautions and local biosafety guidelines for handling and disposal of SKYSONA to avoid potential transmission of infectious diseases. Remove each metal cassette from liquid nitrogen storage. Confirm that SKYSONA is printed on the infusion bag(s) label(s). Confirm that patient identity matches the unique patient identification information located on the SKYSONA infusion bag(s). Do not infuse SKYSONA if the information on the patient-specific label on the infusion bag does not match the intended patient, and contact Genetix Biotherapeutics at 1-833-999-6378. A SKYSONA dose may be contained in one or two patient-specific infusion bags. Ensure that the correct number of infusion bags are present. Use the accompanying Lot Information Sheet to confirm that each infusion bag is within the expiration date prior to preparation of SKYSONA for infusion. Remove the overwrap and inspect each infusion bag for any breaches of integrity before thawing and infusion. If an infusion bag is compromised, follow the local guidelines and contact Genetix Biotherapeutics immediately at 1-833-999-6378. If more than one infusion bag is provided to achieve the treatment dose, thaw and administer each infusion bag completely before proceeding to thaw the next infusion bag. Maintain the second infusion bag, if applicable, within the cold-storage dewar to maintain temperature less than or equal to -140°C (-220°F) until time to thaw. Thaw SKYSONA at 37°C (98.6°F) in a water bath or dry bath. Thawing of each infusion bag takes approximately 2 to 4 minutes. Promptly remove the infusion bag from the bath once thawed. Do not leave SKYSONA unattended and do not submerge the infusion ports if thawed in a water bath. After thawing, mix SKYSONA gently by massaging the infusion bag to disperse clumps of cellular material until all of the contents are uniform. If visible cell clumps remain, continue to gently mix the contents of the bag. Most small clumps of cellular material should disperse with gentle manual mixing. Do not filter, wash, spin down and/or resuspend SKYSONA in new media prior to infusion. Do not sample, alter, irradiate or refreeze SKYSONA. 2.3 Administration SKYSONA is for autologous use only. The patient's identity must match the patient identifiers on the SKYSONA cassette(s) and infusion bag(s). Do not infuse SKYSONA if the information on the patient-specific label does not match the intended patient. Do not use an in-line blood filter or an infusion pump. Before infusion, confirm that the patient's identity matches the unique patient identifiers on the SKYSONA infusion bag(s). The total number of infusion bags to be administered should also be confirmed with the Lot Information Sheet. Prime the tubing of the infusion set with 0.9% sodium chloride solution prior to infusion. Expose the sterile port on the infusion bag by tearing off the protective wrap covering the port. Access the SKYSONA infusion bag and infuse per the treatment center's standard procedures for administration of cell therapy products. Complete the infusion of SKYSONA as soon as possible, and no more than 4 hours after thawing. Administer each infusion bag of SKYSONA via intravenous infusion (drip) by gravity flow over a period of less than 60 minutes. After the entire content of the infusion bag is infused, flush all SKYSONA remaining in the infusion bag and any associated tubing with at least 50 mL of 0.9% sodium chloride solution to ensure that as many cells as possible are infused into the patient. If more than one infusion bag is provided, administer each infusion bag completely before proceeding to thaw (following Section 2.2 steps 7-8) and infuse (following Section 2.3 steps 2-6) the next infusion bag. After SKYSONA Administration Standard procedures for patient management after HSC transplantation should be followed after SKYSONA infusion. Irradiate any blood products required within the first 3 months after SKYSONA infusion. Patients treated with SKYSONA should not donate blood, organs, tissues, or cells at any time in the future.

4 CONTRAINDICATIONS None. None. ( 4 )

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; 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. The most common non-laboratory, serious adverse reactions (≥ 3% incidence) that occurred after treatment with SKYSONA were febrile neutropenia (18%), pyrexia (fever) (18%), seizure (7%), myelodysplastic syndrome (4%), pseudomonal bacteremia (3%), pancytopenia (3%), vascular device infection (3%), mucositis (3%), and vomiting (3%). Most common non-laboratory adverse reactions by time of onset follow: During mobilization and conditioning and occurring in ≥ 20% of patients: nausea (79%), vomiting (72%), decreased appetite (42%), catheter site pain (39%), constipation (30%), headache (24%), abdominal pain (21%), rash (13%) In the first 60 days after treatment with SKYSONA in ≥ 15% of patients: mucositis (88%), febrile neutropenia (73%), alopecia (72%), abdominal pain (33%), vomiting (31%), decreased appetite (31%), pyrexia (27%), nausea (27%), constipation (21%), diarrhea (21%), epistaxis (19%), pruritus (18%), headache (16%), oropharyngeal pain (16%), skin hyperpigmentation (16%), anxiety (15%) Between 60 days and 1 year after treatment with SKYSONA in ≥ 5% of patients: pyrexia (fever) (9%) and vomiting (6%) Table 1 presents non-laboratory adverse reactions reported in at least 10% of patients between the start of conditioning and 24 months after SKYSONA administration. Table 2 presents Grade 3 or 4 laboratory abnormalities that occurred in at least 40% of patients between the start of conditioning and 24 months after SKYSONA administration. Table 1: Non-Laboratory Adverse Reactions Reported in ≥ 10% of Patients Between the Start of Conditioning and 24 Months Following Treatment with SKYSONA Includes adverse events associated with conditioning. Adverse Reaction Any Grade n (%) Grade 3 or Higher n (%) Blood and lymphatic system disorders -- -- Febrile neutropenia Febrile neutropenia includes febrile bone marrow aplasia and febrile neutropenia. 49 (73%) 49 (73%) Cardiac disorders -- -- Tachycardia Tachycardia includes sinus tachycardia and tachycardia. 10 (15%) 0 Eye disorders -- -- Vision blurred 7 (10%) 0 Gastrointestinal disorders -- -- Mucositis Mucositis includes anal inflammation, colitis, gastrointestinal inflammation, mucosal inflammation, proctitis, and stomatitis. Encompasses more than one system organ class. 62 (92%) 34 (51%) Nausea 56 (84%) 17 (25%) Vomiting 51 (76%) 12 (18%) Abdominal pain Abdominal pain includes abdominal discomfort, abdominal pain, and abdominal pain upper. 30 (45%) 2 (3%) Constipation 28 (42%) 0 Diarrhea 19 (28%) 1 (1%) General disorders and administration site conditions -- -- Pyrexia 24 (36%) 3 (4%) Injury, poisoning and procedural complications -- -- Transfusion reaction Transfusion reaction includes allergic transfusion reaction and anaphylactic transfusion reaction. 8 (12%) 2 (3%) Metabolism and nutrition disorders -- -- Decreased appetite 43 (64%) 27 (40%) Nervous system disorders -- -- Headache 19 (28%) 0 Anxiety Anxiety includes akathisia, agitation, anxiety, and irritability. 10 (15%) 0 Respiratory, thoracic, and mediastinal disorders -- -- Epistaxis 13 (19%) 5 (7%) Oropharyngeal pain Oropharyngeal pain includes mouth ulceration, oral pain, and oropharyngeal pain. 12 (18%) 3 (4%) Cough 7 (10%) 0 Skin and subcutaneous tissue disorders -- -- Alopecia 48 (72%) 1 (1%) Rash Rash includes rash, rash erythematous, rash maculo-papular, and urticaria. 14 (21%) 0 Pruritus Pruritus includes anal pruritus, pruritus, and pruritus allergic. 13 (19%) 0 Skin hyperpigmentation 12 (18%) 0 Vascular disorders -- -- Hypertension 8 (12%) 1 (1%) Hematologic Malignancy As of July 2025, hematologic malignancies have been diagnosed in 10/67 (15%) clinical study patients [see Warnings and Precautions (5.1) ] . At diagnosis of hematologic malignancy, all 10 patients had predominant integrations; 7 in proto-oncogenes, including 6 in MECOM . Pathological diagnoses ranged between myelodysplastic syndrome (MDS)-unilineage dysplasia to acute myeloid leukemia. As of July 2025, 9 of the 10 patients had received allogeneic hematopoietic stem cell transplant. One of the patients with MDS relapsed 6 months after an allogeneic transplant and required re-treatment of MDS. Serious Infections Important opportunistic infections that have been diagnosed within the first 3 months after treatment with SKYSONA include BK cystitis, cytomegalovirus reactivation, human herpesvirus-6 viremia, candidiasis, and bacteremias. Opportunistic infections after the first 3 months include an atypical mycobacterium vascular device infection, pseudomonas bacteremia, and Epstein-Barr virus reactivations diagnosed as late as 18 months after treatment with SKYSONA. Serious infections involving adenovirus include a case of transverse myelitis at 6 months that was attributed to adenovirus and entero/rhinovirus infection, and a fatal adenovirus infection at 21 months in a patient with CALD progression who developed multisystem organ failure. Grade 3 or higher infections occurred in 21% of all patients (12% bacterial, 3% viral, and 6% unspecified). The most common Grade 3 or higher infections were vascular device infections (7% of patients) diagnosed as late as 6 months after treatment with SKYSONA, and bacteremias (6% of patients) diagnosed as late as 8 months after treatment with SKYSONA. Table 2: Grade 3 or 4 Laboratory Abnormalities Occurring in ≥ 40% of Patients Between the Start of Conditioning and 24 Months Following Treatment with SKYSONA Includes laboratory abnormalities associated with conditioning. Laboratory Abnormality Grade 3 or 4 n (%) Leukopenia 67 (100%) Lymphopenia 67 (100%) Thrombocytopenia 67 (100%) Neutropenia 64 (96%) Anemia 56 (84%) Hypokalemia 28 (42%) Cytopenias Grade 3 or higher cytopenias on or after Day 60 following SKYSONA infusion occurred in 47% of patients and included low platelet count (14%), low neutrophil count (22%), low lymphocyte count (27%), and low hemoglobin (2%). Grade 3 cytopenias persisted beyond Day 100 in 15% of patients and included low platelet count (7%), low neutrophil count (9%), and low lymphocyte count (6%). Serious adverse reactions of pancytopenia occurred in two patients who required support with blood and platelet transfusions as well as growth factors (G-CSF for up to 6 months and eltrombopag for up to 14 months) after SKYSONA administration. One patient had intercurrent parvovirus infection and his pancytopenia was ongoing at least two years after SKYSONA administration. Pancytopenia in the other patient was ongoing until he was diagnosed with myelodysplastic syndrome approximately two years after SKYSONA administration. Platelet Engraftment Delay Platelet engraftment was defined as 3 consecutive platelet values ≥ 20 × 10 9 /L on different days and no platelet transfusions administered for 7 days immediately preceding and during the evaluation period. Platelet engraftment was not achieved by Day 43 after SKYSONA administration in 13 of 63 patients (21%). Patients treated with SKYSONA achieved platelet engraftment at median (min, max) Day 29 (14, 108) in clinical studies, including two patients treated with a thrombopoietin receptor agonist at the time engraftment criteria were met until 10 or 14 months after treatment with SKYSONA. One of the two had persistence of mild thrombocytopenia after discontinuation of the eltrombopag, and the other remained severely thrombocytopenic (platelet count < 50 × 10 9 /L) until he was diagnosed with myelodysplastic syndrome approximately 2 years after SKYSONA administration [see Warnings and Precautions (5.4) ] . Neutrophil Engraftment Neutrophil engraftment was defined as achieving 3 consecutive absolute neutrophil counts (ANC) ≥ 0.5 × 10 9 cells/L (after initial post-infusion nadir) obtained on different days by Day 43 after SKYSONA infusion. While all patients met criteria for neutrophil engraftment following treatment with SKYSONA in clinical trials, 7 of 67 patients (10%) required G-CSF beyond Day 43, including 3 patients who required G-CSF more than 3 months after treatment with SKYSONA. In three other patients, G-CSF discontinuation was followed by a decrease in neutrophil count to < 0.5 × 10 9 cells/L occurring within 3 days and lasting for two to five weeks [see Warnings and Precautions (5.5) ] . Infusion-Related Reactions Nausea and vomiting have occurred on the day of infusion. Premedication with anti-emetic, anti-pyretics and/or antihistamines may be considered.

What is Skysona used for?
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 …

Who makes Skysona?
Genetix Biotherapeutics Inc. is the labeler of record for Skysona.

What form does Skysona come in?
administered by intravenous. Strengths and pack sizes vary by label, so compare the NDC on your pack with the identifier listed on this page.

Is Skysona prescription or over-the-counter?
The FDA label for this product is recorded as cellular therapy. Marketing status can differ between strengths and countries, so check the dispensing rules where you live.

Has Skysona been recalled?
No FDA recall record in our database matches this product name or manufacturer. Recall data is updated periodically, so a very recent notice may not appear here yet.

Where does this information come from?
All content is reproduced from FDA Structured Product Labelling data via openFDA. It is provided for identification and reference only and is not medical advice.