Ciltacabtagene Autoleucel
/api/v1/drug/ciltacabtagene-autoleucelBoxed warning
CYTOKINE RELEASE SYNDROME, NEUROLOGIC TOXICITIES, HLH/MAS, PROLONGED and RECURRENT CYTOPENIA, and SECONDARY HEMATOLOGICAL MALIGNANCIES Cytokine Release Syndrome (CRS), including fatal or life-threatening reactions, occurred in patients following treatment with CARVYKTI. Do not administer CARVYKTI to patients with active infection or inflammatory disorders. Treat severe or life-threatening CRS with tocilizumab or tocilizumab and corticosteroids [see Dosage and Administration (2.2 , 2.3) , Warnings and Precautions (5.2) ] . Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS), which may be fatal or life-threatening, occurred following treatment with CARVYKTI, including before CRS onset, concurrently with CRS, after CRS resolution, or in the absence of CRS. Monitor for neurologic events after treatment with CARVYKTI. Provide supportive care and/or corticosteroids as needed [see Dosage and Administration (2.2 , 2.3) , Warnings and Precautions (5.3) ] . Parkinsonism and Guillain-Barré syndrome (GBS) and their associated complications resulting in fatal or life-threatening reactions have occurred following treatment with CARVYKTI [see Warnings and Precautions (5.3) ] . Hemophagocytic Lymphohistiocytosis/Macrophage Activation Syndrome (HLH/MAS), including fatal and life-threatening reactions, occurred in patients following treatment with CARVYKTI.
Mechanism of action
Sourced from openFDACARVYKTI is a BCMA-directed, genetically modified autologous T cell immunotherapy, which involves reprogramming a patient's own T cells with a transgene encoding a CAR that identifies and eliminates cells that express BCMA. The CARVYKTI CAR protein features two BCMA-targeting single-domain antibodies designed to confer high avidity against human BCMA, a 4-1BB co-stimulatory domain and a CD3-zeta (CD3ζ) signaling cytoplasmic domain.
Indications
Sourced from openFDA- CARVYKTI is indicated for the treatment of adult patients with relapsed or refractory multiple myeloma, who have received at least 1 prior line of therapy, including a proteasome inhibitor and an immunomodulatory agent, and are refractory to lenalidomide. CARVYKTI is a B-cell maturation antigen (BCMA)-directed genetically modified autologous T cell immunotherapy indicated for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least 1 prior line of therapy, including a proteasome inhibitor and an immunomodulatory agent, and are refractory to lenalidomide.ICD-10: C90.00
Contraindications
Sourced from openFDA- None.contraindicated
Dosage & administration
Sourced from openFDAFor autologous use only. For intravenous use only. Administer a lymphodepleting regimen of cyclophosphamide and fludarabine before infusion of CARVYKTI. ( 2.2 ) Do NOT use a leukodepleting filter. ( 2.2 ) Verify the patient's identity prior to infusion. ( 2.2 ) Premedicate with acetaminophen and an H1-antihistamine. ( 2.2 ) Avoid prophylactic use of systemic corticosteroids. ( 2.2 ) Confirm availability of tocilizumab prior to infusion. ( 2.2 , 5.2 ) Dosing of CARVYKTI is based on the number of chimeric antigen receptor (CAR)-positive viable T cells. ( 2.1 ) Recommended dose range is 0.5–1.0×10 6 CAR-positive viable T cells per kg of body weight, with a maximum dose of 1×10 8 CAR-positive viable T cells per single-dose infusion. ( 2.1 ) 2.1 Dose CARVYKTI is provided as a single dose for infusion containing a suspension of chimeric antigen receptor (CAR)-positive viable T cells in one infusion bag. The recommended dose range is 0.5–1.0×10 6 CAR-positive viable T cells per kg of body weight, with a maximum dose of 1×10 8 CAR-positive viable T cells per single infusion. 2.2 Administration CARVYKTI is for autologous use only. For intravenous use only. The patient's identity must match the patient identifiers on the CARVYKTI cassette and infusion bag. Do not infuse CARVYKTI if the information on the patient-specific labels does not match the intended patient. Preparing the Patient for CARVYKTI Infusion Confirm availability of CARVYKTI prior to starting the lymphodepleting chemotherapy regimen.
Warnings & precautions
Sourced from openFDAProlonged and Recurrent Cytopenias : Patients may exhibit ≥Grade 3 cytopenias following CARVYKTI infusion. One or more recurrences of Grade 3 or higher cytopenias may occur after partial or complete recovery of cytopenias. Monitor blood counts prior to and after CARVYKTI infusion. Prolonged neutropenia has been associated with increased risk of infection. ( 5.5 ) Infections : Monitor patients for signs and symptoms of infection; treat appropriately. ( 5.6 ) Hypogammaglobulinemia : Monitor and consider immunoglobulin replacement therapy. ( 5.7 ) Hypersensitivity Reactions : Hypersensitivity reactions have occurred. Monitor for hypersensitivity reactions during infusion. ( 5.8 ) Immune Effector Cell-associated Enterocolitis: Patients may exhibit prolonged and severe diarrhea in the months following CARVYKTI infusion. Monitor for signs and symptoms of IEC-Enterocolitis after CARVYKTI infusion and manage per institutional guidelines. ( 5.9 ) Secondary Malignancies : Secondary hematological malignancies, including myelodysplastic syndrome and acute myeloid leukemia, have occurred. T-cell malignancies have occurred following treatment of hematologic malignancies with BCMA- and CD19-directed genetically modified autologous T-cell immunotherapies, including CARVYKTI. In the event that a secondary malignancy occurs after treatment with CARVYKTI, contact Janssen Biotech, Inc. at 1-800-526-7736.
Adverse reactions
Sourced from openFDAThe most common nonlaboratory adverse reactions (incidence greater than 20%) are pyrexia, cytokine release syndrome, hypogammaglobulinemia, hypotension, musculoskeletal pain, fatigue, infections-pathogen unspecified, cough, chills, diarrhea, nausea, encephalopathy, decreased appetite, upper respiratory tract infection, headache, tachycardia, dizziness, dyspnea, edema, viral infections, coagulopathy, constipation, and vomiting. The most common Grade 3 or 4 laboratory adverse reactions (incidence greater than or equal to 50%) include lymphopenia, neutropenia, white blood cell decreased, thrombocytopenia, and anemia. ( 6 ) To report SUSPECTED ADVERSE REACTIONS, contact Janssen Biotech, Inc. at 1-800-526-7736 (1-800-JANSSEN) 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 the WARNINGS and PRECAUTIONS section reflect exposure to CARVYKTI in 285 patients with relapsed or refractory multiple myeloma: one randomized, open-label with 188 patients in CARTITUDE-4 and one single-arm, open-label study with 97 patients in CARTITUDE-1.
Use in specific populations
Sourced from openFDA8.1 Pregnancy Risk Summary There are no available data on the use of CARVYKTI in pregnant women. No reproductive and developmental toxicity studies in animals have been conducted with CARVYKTI to assess whether it can cause fetal harm when administered to a pregnant woman. It is not known whether CARVYKTI has the potential to be transferred to the fetus and cause fetal toxicity. Based on the mechanism of action, if the transduced cells cross the placenta, they may cause fetal toxicity, including B-cell lymphocytopenia and hypogammaglobulinemia. Therefore, CARVYKTI is not recommended for women who are pregnant, or for women of childbearing potential not using contraception. Pregnant women should be advised that there may be risks to the fetus. Pregnancy after CARVYKTI therapy 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. 8.2 Lactation Risk Summary There is no information regarding the presence of CARVYKTI in human milk, the effect on the breastfed infant, and the effects on milk production.
Pharmacokinetics
Sourced from openFDA- Metabolism
- The pharmacokinetics (PK) of CARVYKTI was assessed in 285 adult patients with relapsed or refractory multiple myeloma in CARTITUDE-1 and CARTITUDE-4 receiving a single infusion at the median dose of 0.71×10 6 CAR-positive viable T cells/kg (range: 0.41×10 6 to 1.08×10 6 cells/kg). Following a single infusion, CARVYKTI exhibited an initial expansion phase followed by a rapid decline, and then a slower decline.
FAERS reports
- 1Cytokine Release Syndrome71914%
- 2Pyrexia3306.5%
- 3Immune Effector Cell-associated Neurotoxicity Syndrome2905.7%
- 4Hospitalisation2835.6%
- 5Parkinsonism2815.5%
- 6Adverse Event2074.1%
- 7Neurotoxicity1773.5%
- 8Fatigue1703.3%
- 9Death1673.3%
- 10Malaise1663.3%
- 11Cranial Nerve Paralysis1462.9%
- 12Facial Paralysis1302.6%
- 13Bell^s Palsy1162.3%
- 14Neutropenia1132.2%
- 15Illness1092.1%
Clinical trials
The 10 most recently updated of 27 ClinicalTrials.gov registrations naming Ciltacabtagene Autoleucel as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.
- Recombinant Human IL-7 (NT-I7) in Relapsed/Refractory Multiple Myeloma Following BCMA CAR-T Therapy (Cilta-cel)Recruiting · Phase 1 · Interventional · 52 enrolled · Washington University School of MedicineNCT07200089updated 2026-06-10
- Selinexor Maintenance Post CAR-T Cell Therapy for Multiple MyelomaRecruiting · Phase 1 · Interventional · 20 enrolled · Washington University School of MedicineNCT07200102updated 2026-06-08
- A Long-term Study for Participants Previously Treated With Ciltacabtagene AutoleucelRecruiting · Phase 4 · Interventional · 295 enrolled · Janssen Research & Development, LLCNCT05201781updated 2026-06-05
- A Study of Ciltacabtagene Autoleucel and Talquetamab for the Treatment of Participants With High-Risk Multiple MyelomaActive not recruiting · Phase 2 · Interventional · 11 enrolled · Janssen Research & Development, LLCNCT06550895updated 2026-06-05
- A Study of JNJ-68284528, a Chimeric Antigen Receptor T Cell (CAR-T) Therapy Directed Against B-cell Maturation Antigen (BCMA) in Participants With Multiple MyelomaActive not recruiting · Phase 2 · Interventional · 208 enrolled · Janssen Research & Development, LLCNCT04133636updated 2026-06-05
- A Study of Different Sequences of Cilta-cel, Talquetamab in Combination With Daratumumab and Teclistamab in Combination With Daratumumab Following Induction With Daratumumab, Bortezomib, Lenalidomide and Dexamethasone in Participants With Standard-risk Newly Diagnosed Multiple MyelomaActive not recruiting · Phase 2 · Interventional · 43 enrolled · Janssen Research & Development, LLCNCT06577025updated 2026-06-05
- A Study of Bortezomib, Lenalidomide and Dexamethasone (VRd) Followed by Cilta-cel, a CAR-T Therapy Directed Against BCMA Versus VRd Followed by Lenalidomide and Dexamethasone (Rd) Therapy in Participants With Newly Diagnosed Multiple Myeloma for Whom ASCT is Not Planned as Initial TherapyActive not recruiting · Phase 3 · Interventional · 743 enrolled · Janssen Research & Development, LLCNCT04923893updated 2026-06-05
- A Study to Evaluate Efficacy and Safety of Ciltacabtagene AutoleucelRecruiting · Phase 2 · Interventional · 60 enrolled · Janssen Research & Development, LLCNCT07149857updated 2026-06-05
- A Study Comparing JNJ-68284528, a CAR-T Therapy Directed Against B-cell Maturation Antigen (BCMA), Versus Pomalidomide, Bortezomib and Dexamethasone (PVd) or Daratumumab, Pomalidomide and Dexamethasone (DPd) in Participants With Relapsed and Lenalidomide-Refractory Multiple MyelomaActive not recruiting · Phase 3 · Interventional · 419 enrolled · Janssen Research & Development, LLCNCT04181827updated 2026-05-11
- BEAM-MM - β-Hydroxybutyrate-Enhanced Adaptive Immunity in Multiple MyelomaRecruiting · Interventional · 45 enrolled · Universitätsklinikum Hamburg-EppendorfNCT07564219updated 2026-05-04
Frequently asked questions
- How does Ciltacabtagene Autoleucel work?
- CARVYKTI is a BCMA-directed, genetically modified autologous T cell immunotherapy, which involves reprogramming a patient's own T cells with a transgene encoding a CAR that identifies and eliminates cells that express BCMA. The CARVYKTI CAR protein features two BCMA-targeting single-domain antibodies designed to confer high avidity against human BCMA, a 4-1BB co-stimulatory domain and a CD3-zeta (CD3ζ) signaling cytoplasmic domain.
- What is Ciltacabtagene Autoleucel used for?
- According to FDA labeling, Ciltacabtagene Autoleucel carries indications including: CARVYKTI is indicated for the treatment of adult patients with relapsed or refractory multiple myeloma, who have received at least 1 prior line of therapy, including a proteasome inhibitor and an immunomodulatory agent, and are refractory to lenalidomide. CARVYKTI is a B-cell maturation antigen (BCMA)-directed genetically modified autologous T cell immunotherapy indicated for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least 1 prior line of therapy, including a proteasome inhibitor and an immunomodulatory agent, and are refractory to lenalidomide.. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.
- What class of drug is Ciltacabtagene Autoleucel?
- Ciltacabtagene Autoleucel is classified as Antineoplastic cell and gene therapy, Biological Response Modifiers, Cell-mediated Immunity.
- What are the brand names for Ciltacabtagene Autoleucel?
- Ciltacabtagene Autoleucel is marketed under brand names including Carvykti.
- What are the contraindications for Ciltacabtagene Autoleucel?
- Ciltacabtagene Autoleucel labeling lists contraindications including: None.. Always consult the full prescribing information and a clinician.
ciltacabtagene-autoleucel is illustrative MVP content compiled from public sources. pharmacopeia is for educational and informational use only and is not a substitute for professional medical advice.