Ciltacabtagene autoleucel



Ciltacabtagene autoleucel is a BCMA-directed CAR T-cell therapy used in the treatment of relapsed or refractory multiple myeloma in previously treated patients.

Brand Names
Generic Name
Ciltacabtagene autoleucel
DrugBank Accession Number

Multiple myeloma is a malignancy involving the plasma cells of the bone marrow. It is a rare malignancy, with an estimated yearly incidence of 6.5 people per 100,000,4 and is variable in its presentation - some patients may remain entirely asymptomatic, while others may experience a range of symptoms including bone pain, hematologic abnormalities, and end-organ damage.4 There have been a number of treatments developed for multiple myeloma (e.g. daratumumab), although none are curative.1

B-cell maturation antigen (BCMA) is a transmembrane glycoprotein member of the tumor necrosis factor receptor superfamily 17 (TNFRSF17) which is used as a biomarker for multiple myeloma.1 While normally expressed on plasma blasts and plasma cells, BCMA is widely expressed on malignant plasma cells and most multiple myeloma cell lines, making it a choice target in the development of immunotherapies against multiple myeloma.1

Ciltacabtagene autoleucel (Carvykti, Jannsen Biotech Inc.) is a BCMA-directed genetically modified autologous T-cell immunotherapy.2 Patient T-cells are reprogrammed with a transgene encoding a specific chimeric antigen receptor (CAR) which features two BCMA-targeting single-domain antibodies.2 Re-infusion of these modified T-cells leads to the targeted elimination of malignant plasma cells, on which BCMA is highly expressed.1 Carvykti was first approved by the FDA in February 2022 for the treatment of relapsed or refractory multiple myeloma in treatment-experienced patients.3

Approved, Investigational
Biologic Classification
Cell transplant therapies
Autologous cell transplant
  • Autologous bi-epitope BCMA-targeted CAR T-cells
  • Ciltacabtagene autoleucel
External IDs
  • JNJ-68284528
  • LCAR-B38M CAR-T cells



Ciltacabtagene autoleucel is indicated for the treatment of adult patients with relapsed or refractory multiple myeloma after ≥4 prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody.2

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Associated Conditions
Contraindications & Blackbox Warnings
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Treatment with ciltacabtagene autoleucel comprises a single infusion of a dose range between 0.5-1.0x106 CAR-positive viable T-cells per kilogram of body weight.2

Patients receiving treatment with ciltacabtagene autoleucel are required to undergo monitoring through a Risk Evaluation and Mitigation Strategy (REMS) called the Carvykti REMS.2 There are a number of potentially serious adverse reactions related to ciltacabtagene autoleucel therapy which require close monitoring and intervention. Cytokine Release Syndrome (CRS), which may be fatal, may be mitigated with the use of tocilizumab and/or corticosteroids.2 Similarly, significant neurologic toxicities (including Immune Effector Cell-Associated Neurotoxicity Syndrome [ICANS]) may occur and can be treated with supportive care and/or corticosteroid therapy as required.2 Serious hematologic adverse effects - including hemophagocytic lymphohistiocytosis (HLH), macrophage activation syndrome (MAS), and various recurrent/prolonged cytopenias - have also been observed in patients following treatment with ciltacabtagene autoleucel.2

Mechanism of action

Ciltacabtagene autoleucel is a chimeric antigen receptor (CAR) T-cell therapy in which genetically modified autologous T-cells are reprogrammed to target B-cell maturation antigen (BCMA), a biomarker of multiple myeloma.2 Patient peripheral blood mononuclear cells are obtained via leukapheresis, after which they are enriched for T-cells and genetically modified ex vivo to express a CAR comprising an anti-BCMA targeting domain consisting of two single-domain anti-BCMA antibodies linked to a 4-1BB costimulatory domain and a CD3-zeta signaling domain.2

The genetically modified CAR T-cells are then expanded, washed, and cryopreserved for shipping back to the patient. When the product is infused back into the patient, the anti-BCMA CAR T-cells are able to recognize and eliminate BCMA-expressing target cells, including malignant plasma cells involved in multiple myeloma.2

ATumor necrosis factor receptor superfamily member 17

Following a single infusion of a median dose of 0.71x106 CAR positive viable T cells/kg, the median Cmax and AUC0-28d were 47806 copies/µg genomic DNA and 371569 copies*day/µg genomic DNA.2 The median Tmax was 12.7 days.2

Volume of distribution

Not Available

Protein binding

Not Available

Not Available
Route of elimination

Not Available


The median half-life of ciltacabtagene autoleucel following a single infusion of a median dose of 0.71x106 CAR positive viable T cells/kg was 15.3 days.2


Not Available

Adverse Effects
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Not Available

Not Available
Pharmacogenomic Effects/ADRs
Not Available


Drug Interactions
This information should not be interpreted without the help of a healthcare provider. If you believe you are experiencing an interaction, contact a healthcare provider immediately. The absence of an interaction does not necessarily mean no interactions exist.
Not Available
Food Interactions
Not Available


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International/Other Brands
Carvykti (Janssen Biotech, Inc.)
Brand Name Prescription Products
NameDosageStrengthRouteLabellerMarketing StartMarketing EndRegionImage
CarvyktiInjection, suspension100000000 1/1IntravenousJanssen Biotech, Inc2022-02-28Not applicableUS flag
CarvyktiInjection51600000 cellsIntravenousJanssen Cilag International Nv2022-06-21Not applicableEU flag
CarvyktiSuspension100000000 cells / bagIntravenousJanssen PharmaceuticalsNot applicableNot applicableCanada flag


Drug Categories
Not Available
Not classified
Affected organisms
  • Humans

Chemical Identifiers

CAS number
Not Available


General References
  1. Nobari ST, Nojadeh JN, Talebi M: B-cell maturation antigen targeting strategies in multiple myeloma treatment, advantages and disadvantages. J Transl Med. 2022 Feb 10;20(1):82. doi: 10.1186/s12967-022-03285-y. [Article]
  2. FDA Approved Drug Products: Carvykti (ciltacabtagene autoleucel) suspension for intravenous infusion [Link]
  3. Johnson & Johnson: U.S. FDA Approves CARVYKTI™ (ciltacabtagene autoleucel), Janssen’s First Cell Therapy, a BCMA-Directed CAR-T Immunotherapy for the Treatment of Patients with Relapsed or Refractory Multiple Myeloma [Link]
  4. National Organization for Rare Disorders: Multiple Myeloma [Link]

Clinical Trials

Clinical Trials
4RecruitingOtherMultiple Myeloma (MM)1
3Active Not RecruitingTreatmentMultiple Myeloma (MM)1
3Not Yet RecruitingTreatmentMultiple Myeloma (MM)1
3RecruitingTreatmentMultiple Myeloma (MM)1
2RecruitingTreatmentMultiple Myeloma (MM)3
1Not Yet RecruitingTreatmentExtramedullary Myeloma1
1, 2CompletedTreatmentMultiple Myeloma (MM)1
Not AvailableAvailableNot AvailableMultiple Myeloma (MM)1
Not AvailableNo Longer AvailableNot AvailableMultiple Myeloma (MM)1


Not Available
Not Available
Dosage Forms
InjectionIntravenous51600000 cells
Injection, suspensionIntravenous100000000 1/1
SuspensionIntravenous100000000 cells / bag
Not Available
Not Available


Experimental Properties
Not Available


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Pharmacological action
General Function
Receptor for TNFSF13B/BLyS/BAFF and TNFSF13/APRIL. Promotes B-cell survival and plays a role in the regulation of humoral immunity. Activates NF-kappa-B and JNK.
Specific Function
Signaling receptor activity
Gene Name
Uniprot ID
Uniprot Name
Tumor necrosis factor receptor superfamily member 17
Molecular Weight
20165.065 Da
  1. FDA Approved Drug Products: Carvykti (ciltacabtagene autoleucel) suspension for intravenous infusion [Link]

Drug created at November 03, 2021 15:06 / Updated at March 05, 2022 01:02