Elivaldogene autotemcel
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From MedlinePlus, U.S. National Library of Medicine
Boxed warning
WARNING: HEMATOLOGIC MALIGNANCY Hematologic malignancies, including life-threatening cases of myelodysplastic syndrome and acute myeloid leukemia, have occurred in patients treated with SKYSONA. Patients have been diagnosed between 14 months and 10 years after SKYSONA administration, and the cancers appear to be related to treatment with SKYSONA. Monitor patients closely for evidence of malignancy through complete blood counts at least every 3 months. Monitor patients through assessments for evidence for clonal expansion or predominance at least twice in the first year and annually thereafter; consider bone marrow evaluations as clinically indicated [see Warnings and Precautions (5.1) ] .
WARNING: HEMATOLOGIC MALIGNANCY See full prescribing information for complete boxed warning. Hematologic malignancies, including life-threatening cases of myelodysplastic syndrome and acute myeloid leukemia, have occurred in patients treated with SKYSONA. The cancers appear to be the result of treatment with SKYSONA. Monitor patients closely for evidence of malignancy through complete blood counts at least every 3 months and through assessments for evidence for clonal expansion or predominance at least twice in the first year and annually thereafter; consider bone marrow evaluations as clinically indicated. ( 5.1 )
Dosing in kidney impairment
Renal Impairment SKYSONA has not been studied in patients with renal impairment. Patients should be assessed for renal impairment to ensure hematopoietic stem cell (HSC) transplantation is appropriate.
Dosing in liver impairment
Hepatic Impairment SKYSONA has not been studied in patients with hepatic impairment. Patients should be assessed for hepatic impairment to ensure HSC transplantation is appropriate.
Preparation and administration
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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.
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.
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.
Drug interactions
Check elivaldogene autotemcel against a medication listLabel text (3 paragraphs)
Anti-retrovirals : Do not take these medications from at least one month prior to starting mobilization agents until all cycles of apheresis are complete and after the expected duration of elimination. ( 7.2 )
7.1 Vaccines The safety and effectiveness of vaccination during or following SKYSONA treatment have not been studied. Vaccination is not recommended during the 6 weeks preceding the start of myeloablative conditioning, and until hematological recovery following treatment with SKYSONA. Where feasible, administer childhood vaccinations prior to myeloablative conditioning for SKYSONA.
7.2 Anti-retrovirals Patients should not take anti-retroviral medications for at least one month prior to initiating medications for stem cell mobilization and for the expected duration for elimination of the medications, and until all cycles of apheresis are completed [see Warnings and Precautions (5.5) ] . Anti-retroviral medications may interfere with SKYSONA manufacture.
Contraindications
None. None. ( 4 )
Safety data
Reported adverse events (FAERS)
Liver injury (LiverTox)
LiverTox has no chapter for elivaldogene autotemcel. Check the label's hepatic warnings.
Pregnancy and lactation
Pregnancy
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.
For breastfeeding, also see LactMed (National Library of Medicine), which summarizes milk levels and infant effects.
Supply and cost
Drug shortage status
Recalls (last 24 months)
Therapeutic equivalence (Orange Book)
Price ladder
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