elivaldogene autotemcel (Rx)

Brand and Other Names:Skysona
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Dosing & Uses


See Pediatric Dosing

Dosage Forms & Strengths

injection, suspension

  • Each infusion bag contains 20 mL of elivaldogene autotemcel
  • Single dose contains 5 x 106 CD34+ cells/kg of body weight, suspended in a solution containing 5% dimethyl sulfoxide (DMSO)

Cerebral Adrenoleukodystrophy

Indicated to slow the progression of neurologic dysfunction in boys aged 4-17 years with early, active cerebral adrenoleukodystrophy (CALD)

Early, active CALD is defined as 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

Confirm availability and storage of elivaldogene autotemcel and availability of back-up collection of CD34+ cells is confirmed before conditioning

Full myeloablative and lymphodepleting conditioning must be administered before infusion; consult prescribing information for conditioning agents before treatment

After completion of conditioning, allow at least 48 hr of washout before infusion

Elivaldogene autotemcel IV infusion

  • A single dose for infusion containing a suspension of CD34+ cells in 1-2 infusion bags
  • Minimum recommended dose: 5 x 106 CD34+ cells/kg
  • Dose is calculated based on patient’s weight before first apheresis
  • See the Lot Information Sheet provided with product shipment for additional information pertaining to dose

Dosage Modifications

Renal impairment

  • Not studied
  • Assess renal impairment to ensure hematopoietic stem cell (HSC) transplantation is appropriate

Hepatic impairment

  • Not studied
  • Assess for hepatic impairment to ensure HSC transplantation is appropriate

Dosing Considerations

Before therapy

  • Screen for hepatitis B virus (HBV), hepatitis C virus (HCV), human immunodeficiency virus 1 and 2 (HIV-1/HIV-2) and human T-lymphotropic virus 1 and 2 (HTLV-1/HTLV-2) in accordance with clinical guidelines before collecting cells for manufacturing

Mobilization and apheresis

  • Confirm HSC transplantation is appropriate for patient
  • Patients are required to undergo HSC mobilization followed by apheresis to obtain CD34+ cells for product manufacturing
  • Weigh patient before first apheresis collection; collect a minimum target number of CD34+ cells of 12 x 106 CD34+ cells/kg
  • A backup collection of CD34+ cells ≥1.5 x 106 CD34+ cells/kg (if collected by apheresis) or ≥1 x 108 TNC/kg (Total Nucleated Cells, if collected by bone marrow harvest) is required
  • Backup collection may be needed for rescue treatment, if drug is compromised after initiation of conditioning and before its infusion, primary engraftment failure, or loss of engraftment after infusion with this drug
  • Collect and cryopreserve these cells before initiating conditioning and infusion with this product

Limitations of use

  • Does not treat or prevent adrenal insufficiency
  • An immune response to therapy may cause rapid loss of efficacy in patients with full deletions of the human adenosine triphosphate binding cassette, sub family D, member 1 (ABCD1) gene
  • Not studied in CALD secondary to head trauma
  • Owing to the risk of hematologic malignancy, and unclear long-term durability of therapy and human adrenoleukodystrophy protein (ALDP) expression, carefully consider the appropriateness and timing of treatment for each boy, especially for boys with isolated pyramidal tract disease, based on available treatment options since their clinical symptoms do not usually occur until adulthood


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                    Adverse Effects

                    All grades of severity listed unless otherwise indicated


                    During mobilization and conditioning

                    • Nausea (79%)
                    • Vomiting (72%)
                    • Decreased appetite (42%)
                    • Catheter site pain (39%)
                    • Constipation (30%)
                    • Headache (24%)
                    • Abdominal pain (21%)
                    • Rash (13%)
                    • First 60 days after treatment H4
                    • 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%)

                    1 year after treatment

                    • Seizure (15%)

                    Between start of conditioning and 24 months after treatment

                    • Leukopenia (100%)
                    • Lymphopenia (100%)
                    • Thrombocytopenia (100%)
                    • Neutropenia (96%)
                    • Mucositis (92%)
                    • Nausea (84%)
                    • Anemia (84%)
                    • Vomiting (76%)
                    • Febrile neutropenia (73%)
                    • Febrile neutropenia, Grade 3 or more (73%)
                    • Alopecia (72%)
                    • Decreased appetite (64%)
                    • Mucositis, Grade 3 or more (51%)
                    • Abdominal pain (45%)
                    • Constipation (42%)
                    • Hypokalemia (42%)
                    • Decreased appetite, Grade 3 or more (40%)
                    • Diarrhea (28%)
                    • Headache (28%)
                    • Nausea, Grade 3 or more (25%)
                    • Rash (21%)
                    • Epistaxis (19%)
                    • Pruritus (19%)
                    • Vomiting, Grade 3 or more (18%)
                    • Oropharyngeal pain (18%)
                    • Skin hyperpigmentation (18%)
                    • Tachycardia (15%)
                    • Anxiety (15%)
                    • Transfusion reaction (12%)
                    • Hypertension (12%)


                    Between 60 days and 1 year after treatment

                    • Pyrexia (9%)
                    • Vomiting (6%)

                    Between start of conditioning and 24 months after treatment

                    • Vision blurred (10%)
                    • Cough (10%)
                    • Epistaxis, Grade 3 or more (7%)
                    • Oropharyngeal pain, Grade 3 or more (4%)
                    • Abdominal pain, Grade 3 or more (3%)
                    • Transfusion reaction, Grade 3 or more (3%)
                    • Diarrhea, Grade 3 or more (1%)
                    • Alopecia, Grade 3 or more (1%)
                    • Hypertension, Grade 3 or more (1%)


                    Black Box Warnings

                    Hematologic malignancy

                    • Hematologic malignancy, including life-threatening cases of myelodysplastic syndrome, has occurred
                    • Diagnosis was between 14 months and 7.5 years after elivaldogene autotemcel, and cancers appear to be the result of lentiviral vector, Lenti-D, integration in proto-oncogenes
                    • Closely monitor for evidence of malignancy through complete blood counts at least every 6 months and through assessments for evidence of clonal expansion or predominance at least twice in the first year and annually thereafter; consider bone marrow evaluations as clinically indicated




                    May exhibit cytopenias, including pancytopenia, for >1 year following conditioning and infusion; monitor blood cell counts until normalization and assess for signs and symptoms of bleeding and/or infection before and after administration

                    Delayed platelet engraftment has been reported; bleeding risk is increased before platelet engraftment and may continue after engraftment in patients with prolonged thrombocytopenia; monitor for thrombocytopenia and bleeding according to standard guidelines; conduct frequent platelet counts until platelet engraftment and platelet recovery are achieved

                    There is a potential risk of neutrophil engraftment failure after treatment; neutrophil engraftment failure was defined as failure to achieve 3 consecutive absolute neutrophil counts (ANC) ≥0.5 x109 cells/L obtained on different days by Day 43 after infusion; monitor neutrophil counts until engraftment has been achieved; if neutrophil engraftment failure occurs, provide rescue treatment with back-up collection of CD34+ cells

                    Dimethyl sulfoxide (DMSO) may cause hypersensitivity reactions, including anaphylaxis, which is potentially life threatening and requires immediate intervention

                    May affect polymerase chain reaction (PCR) assays for HIV due to LVV provirus insertion; do not use PCR-based assay to screen for HIV infection in treated patients as a false-positive test result is likely

                    Myelodysplastic syndrome (MDS)

                    • MDS, a hematologic malignancy, has developed in treated patients
                    • Carefully consider alternative therapies before treating
                    • Consider consultation with hematology experts before treatment to inform benefit-risk treatment decision and to ensure adequate monitoring for hematologic malignancy
                    • Monitor patients treated with lifelong therapy for hematologic malignancy
                    • For the first 15 years after treatment, monitor via complete blood count (with differential) at least twice per year and via integration site analysis or other testing for evidence of clonal expansion and predominance at least twice in the first year and then annually thereafter
                    • If hematologic malignancy is detected, contact bluebird bio at 1-833-999-6378 for reporting and to obtain instructions on collection of samples for further testing
                    • Consider appropriate expert consultation and additional testing in the following:
                      • Delayed or failed engraftment of platelets or other cell lines (patients who do not achieve unsupported platelet counts ≥20 x 109/L on or after Day 60 appear to be at particularly high risk for developing malignancy), or
                      • New or prolonged cytopenias, or
                      • Presence of clonal expansion or predominance (eg, increasing relative frequency of an integration site, especially if ≥10% and present in MECOM or another proto-oncogene known to be involved in hematologic malignancy)

                    Serious infections

                    • Severe infections, including life-threatening or fatal infections, have occurred
                    • Opportunistic infections (eg, BK cystitis, cytomegalovirus reactivation, human herpesvirus-6 viremia, candidiasis, bacteremias) that have been diagnosed within the first 3 months after treatment
                    • 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
                    • If patient has febrile neutropenia, evaluate for infection, and manage with broad-spectrum antibiotics, fluids, and other supportive care as medically indicated
                    • Monitor for signs and symptoms of infection before and after administration and treat appropriately
                    • Administer prophylactic antimicrobials according to best clinical practices and clinical guidelines
                    • Avoid administration in patients with active infections

                    Use of antiretroviral therapy (ART)

                    • Patients should not take ART for at least 1 month before mobilization or expected duration for elimination of the medications, and until all cycles of apheresis are completed
                    • ART may interfere with manufacturing of the apheresed cells
                    • If a patient requires ART for HIV prophylaxis, delay mobilization and apheresis of CD34+ cells until HIV infection is adequately ruled out


                    • Safety and effectiveness of vaccination during or following treatment not studied
                    • Vaccination is not recommended for 6 weeks preceding the start of myeloablative conditioning, and until hematological recovery following elivaldogene autotemcel
                    • Where feasible, administer childhood vaccinations before myeloablative conditioning

                    Pregnancy & Lactation


                    There are no available data with administration in pregnant females

                    Consider risks associated with mobilization and conditioning agents on pregnancy and fertility

                    No animal reproductive and developmental toxicity studies have been conducted to assess whether fetal harm may occur when administered

                    No nonclinical germline transmission studies have been conducted


                    • Consult prescribing information of mobilization and conditioning agents for information on need for effective contraception
                    • There are insufficient exposure data to provide a precise recommendation on duration of contraception following treatment
                    • Males capable of fathering a child and their female partners of childbearing potential: Use effective method of contraception (intrauterine device or combination of hormonal and barrier contraception) from start of mobilization through at least 6 months after elivaldogene autotemcel


                    • There are no data on effects on fertility
                    • Data are available on the risk of infertility with myeloablative conditioning
                    • Advise patients of option to cryopreserve semen before treatment if appropriate


                    There is no information regarding the presence of elivaldogene autotemcel in human milk, effect on breastfed infants, and effects on milk production

                    Pregnancy Categories

                    A: Generally acceptable. Controlled studies in pregnant women show no evidence of fetal risk.

                    B: May be acceptable. Either animal studies show no risk but human studies not available or animal studies showed minor risks and human studies done and showed no risk.

                    C: Use with caution if benefits outweigh risks. Animal studies show risk and human studies not available or neither animal nor human studies done.

                    D: Use in LIFE-THREATENING emergencies when no safer drug available. Positive evidence of human fetal risk.

                    X: Do not use in pregnancy. Risks involved outweigh potential benefits. Safer alternatives exist.

                    NA: Information not available.



                    Mechanism of Action

                    CALD is a rare, X-linked metabolic disorder caused by mutations in the ABCD1 gene, which affects production of adrenoleukodystrophy protein (ALDP)

                    Elivaldogene autotemcel is administered as a one-time gene therapy designed to add functional copies of the ABCD1 gene into a patient’s own hematopoietic stem cells, resulting in the production of ALDP



                    IV Preparation

                    Coordinate timing of thawing and infusion; confirm infusion time in advance and adjust

                    start time of thawing such that it will be available for infusion when patient and healthcare providers are ready

                    Drug contains human blood cells that are genetically modified with replication-incompetent, self-inactivating Lenti-D lentiviral vector

                    Follow universal precautions and local biosafety guidelines for handling and disposal of elivaldogene autotemcel to avoid potential transmission of infectious diseases

                    Remove each metal cassette from liquid nitrogen storage

                    Confirm that elivaldogene autotemcel is printed on the infusion bag(s) label(s)

                    Confirm that patient identity matches the unique patient identification information located on infusion bag(s)

                    Do not infuse if information on patient-specific label on infusion bag does not match intended patient, and contact bluebird bio at 1-833-999-6378

                    Dose may be contained in 1 or 2 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 before preparation for infusion

                    Remove overwrap and inspect each infusion bag for any breaches of integrity before thawing and infusion

                    If a bag is compromised, follow the local guidelines and contact bluebird bio immediately at 1-833-999-6378

                    Thaw infusion bag at 37ºC (98.6ºF) in a water bath or dry bath, which may take ~2-4 minutes

                    Promptly remove infusion bag from bath once thawed

                    Do not leave bag unattended and do not submerge the infusion ports if thawed in a water bath

                    After thawing, massage infusion bag to gently mix and disperse clumps of cellular material until all the contents are uniform

                    If visible cell clumps remain, continue to gently mix the contents of the bag

                    Do not filter, wash, spin down and/or resuspend elivaldogene autotemcel in new media before infusing

                    Do not sample, alter, irradiate, or refreeze

                    IV Administration

                    Autologous use only

                    Patient’s identity must match patient identifiers on cassette(s) and infusion bag(s)

                    Do not infuse if information on patient-specific label does not match intended patient

                    Do NOT use an inline blood filter or an infusion pump

                    Prime tubing of infusion set with 0.9% NaCl before infusing

                    Tear off the protective wrap on infusion bag to expose sterile port

                    Access infusion bag and infuse per treatment center’s standard procedures for administration of cell therapy products

                    Complete infusion as soon as possible, and ≤4 hr after thawing

                    Administer each infusion bag of IV infusion (drip) by gravity flow over a period of less than 60 minutes

                    After the entire content of the infusion bag is infused, flush remnants in infusion bag and any associated tubing with at least 50 mL of 0.9% NaCl to ensure that as many cells as possible are infused into patient

                    If >1 infusion bag is provided, administer each infusion bag completely before proceeding to thaw and infuse next infusion bag like in the above steps

                    Maintain second infusion bag, if applicable, within cold-storage dewar to maintain temperature ≤-140ºC (-220ºF) until time to thaw

                    After administering elivaldogene autotemcel, standard procedures for patient management after HSC transplantation should be followed

                    Irradiate any blood products required within first 3 months after elivaldogene autotemcel

                    Treated patients should not donate blood, organs, tissues, or cells at any time in the future


                    Frozen product

                    • Store in vapor phase of liquid nitrogen at ≤-140ºC (-220ºF) until ready for thawing and administration
                    • Keep infusion bag(s) in metal cassette(s) and transfer the drug from vapor phase of liquid nitrogen shipper to treatment center vapor phase of liquid nitrogen storage at ≤-140ºC (-220ºF) until ready for thaw and administration
                    • Thaw before infusion
                    • Do not refreeze after thawing
                    • Do not irradiate as this could lead to inactivation

                    Thawed infusion bag

                    • Complete elivaldogene autotemcel as soon as possible, and ≤4 hr after thawing


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                    Patient Handout

                    A Patient Handout is not currently available for this monograph.


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                    Tier Description
                    1 This drug is available at the lowest co-pay. Most commonly, these are generic drugs.
                    2 This drug is available at a middle level co-pay. Most commonly, these are "preferred" (on formulary) brand drugs.
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