Etuvetidigene autotemcel
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From MedlinePlus, U.S. National Library of Medicine
Dosing in kidney impairment
This label has no separate kidney-impairment dosing section. Check warnings and clinical pharmacology in the full label.
Preparation and administration
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Patient Preparation before WASKYRA ® Infusion Mobilization and apheresis Mobilize hematopoietic stem and progenitor cells (HSPC) using G-CSF with plerixafor according to established protocols. Perform apheresis to collect CD34+ cells required for WASKYRA ® manufacturing following the mobilization procedure. Collect and cryopreserve a back-up supply of CD34 + stem cells containing at least 3 x 10⁶ CD34 + cells/kg from the patient.
Complete this collection before initiating reduced intensity conditioning and WASKYRA ® infusion. Store the back-up collection for potential rescue treatment in the following scenarios: WASKYRA ® compromise after reduced intensity conditioning initiation but before scheduled infusion, primary engraftment failure, or prolonged bone marrow aplasia following WASKYRA ® treatment.
Pre-treatment and conditioning Administer rituximab (anti-CD20 monoclonal antibody) approximately 22 days before WASKYRA ® administration to deplete autoreactive B-cells and provide pre-emptive treatment for potential lymphoproliferative disorder due to Epstein Barr Virus infection which is a risk factor in WAS patients.
Administer busulfan and fludarabine for reduced intensity conditioning before infusion of WASKYRA ® to promote engraftment of the genetically modified autologous CD34 + cells. Do not begin conditioning until the complete set of infusion bag(s) constituting the dose of WASKYRA ® has been received and stored at the administration site.
Confirm availability of the back-up collection. Premedication Administer intravenous chlorpheniramine (0.2 mg/kg, max. dose 10 mg), or an equivalent 15-30 minutes before the infusion of WASKYRA ® to reduce the possibility of an allergic reaction to the infusion.
Administration Receipt WASKYRA ® is shipped in the vapor phase of liquid nitrogen (<-130°C; -202°F) from the manufacturing site to the qualified treatment center for the infusion. The Lot Information Sheet and the Chain of Custody documentation travels with the cryoshipper. Upon arrival check the temperature on the display of the cryoshipper, open it using the proper DPI, and confirm the patient code, batch number and number of bags against product label(s).
Transfer WASKYRA ® from the vapor phase of liquid nitrogen at less than -130°C (-202°F) to the treatment center vapor phase of liquid nitrogen storage (<-130°C; -202°F) until ready for thaw and administration. In the event of issues, contact Fondazione Telethon ETS at: 1-888-212-6928. Preparation for infusion WASKYRA contains human blood cells that are genetically modified with a replication-incompetent, self-inactivating lentiviral vector (LVV).
Follow universal precautions and local biosafety guidelines for handling and disposal of WASKYRA ® to avoid potential transmission of infectious diseases. Checking prior to thawing Do not remove the metal cassette from cryogenic storage or thaw WASKYRA ® until the patient is ready to be infused. Coordinate the timing of WASKYRA thaw and infusion.
Confirm the infusion time in advance and adjust the start time for thaw so that the treatment is available for infusion when the patient is ready. Ensure the correct number of infusion bags are present. When more than one bag of WASKYRA ® is provided, thaw one bag of WASKYRA ® at a time. If more than one infusion bag is provided, thaw and administer each infusion bag completely before proceeding to thaw the next infusion bag.
Open the metal cassette and inspect the overwrap bag and infusion bag for any breaches of integrity before thawing. If an infusion bag is compromised, follow the local guidelines for handling of waste of human-derived material and contact Marketing Authorization Holder immediately. Prior to thawing WASKYRA ® , it must be verified that the patient identity matches the unique patient information reported on the packaging labels and Lot Information Sheet (LIS).
WASKYRA ® is solely for autologous use. Do not thaw or infuse WASKYRA ® if the information on the patient-specific label on the infusion bag does not match the intended patient. Confirm that the infusion bag is within the expiration date. Thawing After carefully removing from the metal cassette, thaw the infusion bag in its sealed overwrap bag at 37°C (98.6°F) in a controlled thawing device until there is no visible ice in the infusion bag.
Once thawing is complete, the bag should be removed immediately from the thawing device. The overwrap bag should be carefully opened to remove the infusion bag which should be kept at room temperature (20°C – 25°C; 68°F – 77°F) until infusion. Gently massage the infusion bag to resuspend the cells. The content of the infusion bag should be inspected for any remaining visible cellular aggregates.
Small clumps of cellular material should disperse with gentle manual mixing. Do not shake the bag. To maintain product viability, as soon as possible after thawing is complete, it is recommended that WASKYRA ® is administered immediately. Note: Do not sample, alter, irradiate or refreeze WASKYRA ® . Administration Prior to WASKYRA ® infusion, confirm that the patient’s identity matches the essential unique patient information on the infusion bag(s) labels.
Administer WASKYRA ® as an intravenous infusion using a central venous catheter, per the qualified treatment site’s standard procedures for cell therapy products. When more than one bag of WASKYRA ® is needed, prior to infusion ensure that the volume of WASKYRA ® to be infused is compatible with the recommended limit of dimethyl sulfoxide (DMSO), i.e., the total volume of DMSO administered should remain < 1% of the patient’s estimated plasma volume.
The maximum volume of WASKYRA ® to be administered should therefore remain < 20% of the patient’s estimated plasma volume. Ensure that the administration set consists of a blood transfusion set equipped with a 200 µm filter. Infuse each bag within 2 hours of thaw, including any interruption during the infusion, to maintain maximum product viability.
Infuse contents of each bag within 30 minutes with a maximum infusion rate of 5 mL/kg/h. At the end of the infusion, flush all WASKYRA ® remaining in the infusion bag and any associated tubing with sodium chloride 9 mg/mL (0.9%) solution for injection to ensure that as many cells as possible are infused into the patient.
Consider the infusion volume in relation to the age and weight of the patient. Monitor vital signs (blood pressure, heart rate, and oxygen saturation) and the occurrence of any symptom prior to the start of the infusion, approximately every ten minutes during the infusion and every hour, for 3 hours, after the infusion.
Drug interactions
Check etuvetidigene autotemcel against a medication listLabel text (3 paragraphs)
No formal drug interaction studies have been performed. WASKYRA ® is not expected to interact with the hepatic cytochrome P-450 family of enzymes or drug transporters.
7.1 Effect of Vaccines on WASKYRA ® The safety and effectiveness of vaccination during or following WASKYRA ® treatment have not been studied. Vaccination with live virus vaccines is not recommended until immune reconstitution is completed following treatment with WASKYRA ® .
7.2 Effect of Anti-retrovirals on WASKYRA ® Anti-retroviral medications may interfere with the manufacturing of WASKYRA ® . Patients should not take anti-retroviral medications for at least one month prior to mobilization until at least 7 days after WASKYRA ® infusion. If a patient requires anti-retroviral treatment following exposure to HIV/HTLV, initiation of WASKYRA ® treatment should be delayed until an HIV/HTLV western blot and viral load assay have been performed at 6 months post-exposure.
Contraindications
Hypersensitivity to the active substance or to any of the excipients [see Hypersensitivity and Infusion Related Reactions (5.1) ]. Previous treatment with HSCT within 6 months prior to treatment or HSCT with evidence of residual donor cells. Previous treatment with hematopoietic stem cell gene therapy. Contraindications to the mobilization and the conditioning regimen. Hypersensitivity to the active substance or to any of the excipients. ( 4 ) Previous treatment with HSCT within 6 months prior to screening or HSCT with evidence of residual donor cell. ( 4 ) Previous treatment with hematopoietic stem cell gene therapy. ( 4 ) Contraindications to the mobilization and the conditioning regimen.
( 4 )
Safety data
Reported adverse events (FAERS)
Liver injury (LiverTox)
LiverTox has no chapter for etuvetidigene autotemcel. Check the label's hepatic warnings.
Pregnancy and lactation
Pregnancy
8.1 Pregnancy Risk Summary There are no clinical data from the use of WASKYRA ® in pregnant women. No animal reproductive and developmental toxicity studies have been conducted with WASKYRA ® to assess whether it can cause fetal harm when administered to a pregnant woman. WASKYRA ® must not be administered during pregnancy because of the risk associated with conditioning. Pregnancy after WASKYRA ® infusion should be discussed with the treating physician. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2-4% and 15-20%, 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
Not the price at the counterThese are not what a patient pays at the pharmacy counter. They are what pharmacies, health plans, and government buyers pay, before manufacturer rebates. A patient's cost depends on their insurance (copay, coinsurance, or deductible) or the pharmacy's cash price.