Elivaldogene Autotemcel

Elivaldogene Autotemcel is a prescription medication indicated to slow the progression of neurologic dysfunction in boys aged 4-17 years with early, active cerebral adrenoleukodystrophy (CALD).

• Elivaldogene Autotemcel is available under the following different brand names: Skysona

Common side effects of Elivaldogene Autotemcel include:

• inflammation of mucus membranes
• nausea
• vomiting
• febrile neutropenia
• hair loss
• decreased appetite
• abdominal pain
• constipation
• fever
• diarrhea
• headache
• rash
• low white blood cell count (leukopenia, lymphopenia, neutropenia)
• low platelets (thrombocytopenia)
• low iron (anemia)
• low potassium (hypokalemia)

Serious side effects of Elivaldogene Autotemcel include:

• hives
• difficulty breathing
• swelling of the face, lips, tongue, or throat
• chills
• sweating
• sores in the mouth
• pain when urinating
• increased urination
• sores or pain around the anus
• cough
• shortness of breath
• unusual vaginal discharge or itching
• fever
• seizure
• fatigue
• easy bruising
• pale skin
• pain in the ears and discharge
• skin rash
• pus-filled pimples
• pain, redness and swelling of the eyes
• joint pain and swelling
• neck or back pain
• pus or discharge from a wound
• headache
• diarrhea
• severe coughing
• congestion
• painful urination
• frequent urination
• fast heartbeat
• purple or red spots on the skin
• abnormal bleeding
• vascular device infection
• red or swollen gums
• trouble swallowing
• dry mouth
• white patches in mouth
• increased or thicker saliva in the mouth
• vomiting
• occurring more than 1 year after the last treatment:
• seizure
• shortness of breath
• fatigue
• easy bruising
• paleness

Rare side effects of Elivaldogene Autotemcel  include:

• none 

Seek medical care or call 911 at once if you have the following serious side effects:

• Severe headache, confusion, slurred speech, arm or leg weakness, trouble walking, loss of coordination, feeling unsteady, very stiff muscles, high fever, profuse sweating, or tremors
• Serious eye symptoms such as sudden vision loss, blurred vision, tunnel vision, eye pain or swelling, or seeing halos around lights
• Serious heart symptoms include fast, irregular, or pounding heartbeats; fluttering in the chest; shortness of breath; sudden dizziness, light-headedness, or passing out

This is not a complete list of side effects and other serious side effects or health problems that may occur as a result of the use of this drug. Call your doctor for medical advice about serious side effects or adverse reactions. You may report side effects or health problems to FDA at 1-800-FDA-1088.

Pediatric dosage

Injection, suspension

• Each infusion bag contains 20 mL of Elivaldogene Autotemcel
• Single dose contains 5 × 106 CD34+ cells/kg of body weight, suspended in a solution containing 5% dimethyl sulfoxide

Cerebral adrenoleukodystrophy (CALD)

Pediatric dosage

• Early, active CALD is defined as asymptomatic or mildly symptomatic (neurologic function score of 1 and more) boys who have gadolinium enhancement on brain MRI and Loes scores of 0.5-9
• Confirm availability and storage of Elivaldogene Autotemcel and availability of backup 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 h of washout before infusion

Elivaldogene Autotemcel IV infusion

• A single dose for infusion containing a suspension of CD34+ cells in one to two infusion bags
• Minimum recommended dose: 5 × 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 Considerations – Should be Given as Follows: 

• See “Dosages”

If your medical doctor is using this medicine to treat your pain, your doctor or pharmacist may already be aware of any possible drug interactions and may be monitoring you for them. Do not start, stop, or change the dosage of any medicine before checking with your doctor, healthcare provider, or pharmacist first.

• Elivaldogene Autotemcel has severe interactions with no other drugs.
• Elivaldogene Autotemcel has serious interactions with at least 99 drugs.
• Elivaldogene Autotemcel has moderate interactions with no other drugs.
• Elivaldogene Autotemcel has minor interactions with no other drugs.

This information does not contain all possible interactions or adverse effects. Visit the RxList Drug Interaction Checker for any drug interactions. Therefore, before using this product, tell your doctor or pharmacist about all the products you use. Keep a list of all your medications with you and share this information with your doctor and pharmacist. Check with your healthcare professional or doctor for additional medical advice, or if you have health questions or concerns.

Contraindications

• None

Effects of drug abuse

• None

Short-Term Effects

• See “What Are Side Effects Associated with Using Elivaldogene Autotemcel?”

Long-Term Effects

• See “What Are Side Effects Associated with Using Elivaldogene Autotemcel?”

Cautions

• May exhibit cytopenias, including pancytopenia, for more than 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 for neutrophil engraftment failure after treatment; neutrophil engraftment failure was defined as failure to achieve 3 consecutive absolute neutrophil counts of 0.5 ×109 cells/L and more 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 backup collection of CD34+ cells
• Dimethyl sulfoxide may cause hypersensitivity reactions, including anaphylaxis, which is potentially life-threatening and requires immediate intervention
• May affect polymerase chain reaction (PCR) assays for HIV owing 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 of 20 × 109/L and more on or after day 60 appear to be at particularly high risk of developing malignancy),
  • New or prolonged cytopenias, or
  • Presence of clonal expansion or predominance (eg, increasing relative frequency of an integration site, especially if 10% and more 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 a 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
• Vaccines
  • 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 and Lactation

• There are no available data with administration in pregnant women
• 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
• Contraception
  • 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
  • Men 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
• Infertility
  • There are no data on effects on fertility
  • Data are available on the risk for infertility with myeloablative conditioning
  • Advise patients of the option to cryopreserve semen before treatment if appropriate
• Lactation
  • There is no information regarding the presence of Elivaldogene Autotemcel in human milk, its effect on breastfed infants, and milk production