Tisagenlecleucel

KYMRIAH is a CD19-directed genetically modified autologous T cell immunotherapy which involves reprogramming a patient’s own T cells with a transgene encoding a chimeric antigen receptor (CAR) to identify and eliminate CD19-expressing malignant and normal cells. The CAR is comprised of a murine single-chain antibody fragment which recognizes CD19 and is fused to intracellular signaling domains from 4-1BB (CD137) and CD3 zeta.

• KYMRIAH is a CD19-directed genetically modified autologous T cell immunotherapy indicated for the treatment of: Patients up to 25 years of age with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse. ( 1.1 ) Adult patients with relapsed or refractory (r/r) large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, high grade B-cell lymphoma and DLBCL arising from follicular lymphoma.ICD-10: C85.90, C95.90

• None. None.contraindicated

For autologous use only. For intravenous use only. Administer a lymphodepleting regimen if needed before infusion of KYMRIAH. ( 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 ) Confirm availability of tocilizumab prior to infusion. ( 2.2 , 5.1 ) Dosing of KYMRIAH is based on the number of chimeric antigen receptor (CAR)-positive viable T cells. Pediatric and Young Adult B-cell ALL (up to 25 years of age) • For patients 50 kg or less, administer 0.2 to 5.0 x 10 6 CAR-positive viable T cells per kg body weight intravenously. ( 2.1 ) • For patients above 50 kg, administer 0.1 to 2.5 x 10 8 total CAR-positive viable T cells (non-weight based) intravenously. ( 2.1 ) Adult Relapsed or Refractory Diffuse Large B-cell Lymphoma and Follicular Lymphoma • Administer 0.6 to 6.0 x 10 8 CAR-positive viable T cells intravenously. ( 2.1 ) Note: The patient identifier number may be preceded by the letters DIN or Aph ID. Figure 1. KYMRIAH Infusion Bag 2.1 Recommended Dose For autologous use only. For intravenous use only. Pediatric and Young Adult Relapsed or Refractory B-cell Acute Lymphoblastic Leukemia KYMRIAH is provided as a single-dose for infusion containing a suspension of chimeric antigen receptor (CAR)-positive viable T cells. Based on the patient weight reported at the time of leukapheresis: - Patients 50 kg or less: administer 0.2 to 5.0 x 10 6 CAR-positive viable T cells per kg body weight.

Hypersensitivity Reactions: Monitor for hypersensitivity reactions during infusion. ( 5.4 ) Serious Infections: Monitor patients for signs and symptoms of infection; treat appropriately. ( 5.5 ) Prolonged Cytopenias: Patients may exhibit ≥ Grade 3 cytopenias for several weeks following KYMRIAH infusion. Prolonged neutropenia has been associated with increased risk of infection. ( 5.6 ) Hypogammaglobulinemia: Monitor and provide replacement therapy until resolution. Assess immunoglobulin levels in newborns of mothers treated with KYMRIAH. ( 5.7 ) Secondary Malignancies: T cell malignancies have occurred following treatment of hematologic malignancies with BCMA- and CD19-directed genetically modified autologous T cell immunotherapies, including KYMRIAH. In the event that a secondary malignancy occurs after treatment with KYMRIAH, contact Novartis Pharmaceuticals Corporation at 1-844-4KYMRIAH. ( 5.8 ) 5.1 Cytokine Release Syndrome Cytokine release syndrome (CRS), including fatal or life-threatening reactions, occurred following treatment with KYMRIAH. CRS occurred in 61 (77%) of the 79 pediatric and young adult patients with r/r ALL receiving KYMRIAH, including ≥ Grade 3 CRS (Penn grading system 1 ) occurring in 48% of patients. The median times to onset and resolution of CRS were 3 days (range, 1 to 22; 1 patient with onset after Day 10) and 8 days (range, 1 to 36), respectively. Of the 61 patients with CRS, 31 (51%) received tocilizumab.

Pediatric and Young Adult B-cell ALL (up to 25 years of age): The most common adverse reactions (incidence greater than 20%) are CRS, infections-pathogen unspecified, hypogammaglobulinemia, fever, decreased appetite, viral infectious disorders, headache, febrile neutropenia, hemorrhage, musculoskeletal pain, vomiting, encephalopathy, diarrhea, hypotension, cough, nausea, bacterial infectious disorders, pain, hypoxia, tachycardia, edema, fatigue, and acute kidney injury. ( 6.1 ) Adult Relapsed or Refractory Diffuse Large B-cell Lymphoma: The most common adverse reactions (incidence greater than 20%) are CRS, infections-pathogen unspecified, fever, diarrhea, nausea, fatigue, hypotension, edema, hemorrhage, dyspnea, and headache. ( 6.1 ) Adult Relapsed or Refractory Follicular Lymphoma: The most common adverse reactions (incidence greater than 20%) are CRS, infections-pathogens unspecified, fatigue, musculoskeletal pain, headache, and diarrhea. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Novartis Pharmaceuticals Corporation at 1-888-669-6682 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.

8.1 Pregnancy Risk Summary There are no available data with KYMRIAH use in pregnant women. No animal reproductive and developmental toxicity studies have been conducted with KYMRIAH to assess whether it can cause fetal harm when administered to a pregnant woman. It is not known if KYMRIAH has the potential to be transferred to the fetus. Based on the mechanism of action, if the transduced cells cross the placenta, they may cause fetal toxicity, including B-cell lymphocytopenia. Therefore, KYMRIAH is not recommended for women who are pregnant, and pregnancy after KYMRIAH administration should be discussed with the treating physician. Report pregnancies to Novartis Pharmaceuticals Corporation at 1-888-669-6682. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively. 8.2 Lactation Risk Summary There is no information regarding the presence of KYMRIAH in human milk, the effect on the breastfed infant, and the effects on milk production. A risk to the breastfed infant cannot be excluded. The developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for KYMRIAH and any potential adverse effects on the breastfed infant from KYMRIAH or from the underlying maternal condition.

1. 1Cytokine Release Syndrome1,70938%
2. 2Pyrexia1,03423%
3. 3Malignant Neoplasm Progression59913%
4. 4Immune Effector Cell-associated Neurotoxicity Syndrome4419.9%
5. 5Hypotension4329.7%
6. 6Neurotoxicity4189.4%
7. 7Platelet Count Decreased4139.3%
8. 8White Blood Cell Count Decreased4029.0%
9. 9Hypogammaglobulinaemia3948.8%
10. 10Lymphocyte Count Decreased3718.3%
11. 11Acute Lymphocytic Leukaemia Recurrent3708.3%
12. 12Neutrophil Count Decreased3698.3%
13. 13Haemoglobin Decreased3628.1%
14. 14Diffuse Large B-cell Lymphoma3157.1%
15. 15Death2936.6%

The 10 most recently updated of 45 ClinicalTrials.gov registrations naming Tisagenlecleucel as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• Tisagenlecleucel in Adult Patients With Aggressive B-cell Non-Hodgkin Lymphoma
  Completed · Phase 3 · Interventional · 330 enrolled · Novartis Pharmaceuticals
  NCT03570892updated 2026-06-12
• A Non-Interventional Study PASS to Characterize Secondary Malignancies of Tcell Origin Following Tisagenlecleucel Therapy
  Recruiting · Observational · 30 enrolled · Novartis Pharmaceuticals
  NCT07378969updated 2026-06-11
• Study of Out of Specification for Tisagenlecleucel
  Recruiting · Phase 3 · Interventional · 200 enrolled · Novartis Pharmaceuticals
  NCT04094311updated 2026-06-11
• Outcomes in Pediatric and Young Adult B-Cell Malignancies After Commercially Available Immunotherapy
  Recruiting · Observational · 500 enrolled · Stanford University
  NCT05865301updated 2026-06-04
• Study of Efficacy and Safety of Tisagenlecleucel in HR B-ALL EOC MRD Positive Patients
  Active not recruiting · Phase 2 · Interventional · 121 enrolled · Novartis Pharmaceuticals
  NCT03876769updated 2026-06-02
• Long-term Follow up Local Registry Study of Kymriah in South Korea
  Recruiting · Observational · 500 enrolled · Novartis Pharmaceuticals
  NCT06785818updated 2026-05-05
• Managed Access Programs for CTL019, Tisagenlecleucel
  Available · Expanded access · Novartis Pharmaceuticals
  NCT03601442updated 2026-04-23
• A Phase III Trial Comparing Tisagenlecleucel to Standard of Care (SoC) in Adult Participants With r/r Follicular Lymphoma
  Active not recruiting · Phase 3 · Interventional · 109 enrolled · Novartis Pharmaceuticals
  NCT05888493updated 2026-04-17
• Autologous CD22 CAR T Cells Following Commercial CD19 CAR T Cells in B Cell Malignancies
  Recruiting · Phase 1 · Interventional · 20 enrolled · Stanford University
  NCT06408194updated 2026-02-10
• A Second Infusion (Early Reinfusion) of Tisagenlecleucel in Children and Young Adults With B-Cell Acute Lymphoblastic Leukemia(B-ALL)
  Active not recruiting · Phase 2 · Interventional · 30 enrolled · Memorial Sloan Kettering Cancer Center
  NCT05460533updated 2026-02-04

Frequently asked questions

How does Tisagenlecleucel work?

KYMRIAH is a CD19-directed genetically modified autologous T cell immunotherapy which involves reprogramming a patient’s own T cells with a transgene encoding a chimeric antigen receptor (CAR) to identify and eliminate CD19-expressing malignant and normal cells. The CAR is comprised of a murine single-chain antibody fragment which recognizes CD19 and is fused to intracellular signaling domains from 4-1BB (CD137) and CD3 zeta.

What is Tisagenlecleucel used for?

According to FDA labeling, Tisagenlecleucel carries indications including: KYMRIAH is a CD19-directed genetically modified autologous T cell immunotherapy indicated for the treatment of: Patients up to 25 years of age with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse. ( 1.1 ) Adult patients with relapsed or refractory (r/r) large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, high grade B-cell lymphoma and DLBCL arising from follicular lymphoma.. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Tisagenlecleucel?

Tisagenlecleucel is classified as Antineoplastic cell and gene therapy, CD19-directed Chimeric Antigen Receptor, Genetically-modified Autologous T Cells, CD19 Receptor Interactions, Increased T Lymphocyte Activation.

What are the brand names for Tisagenlecleucel?

Tisagenlecleucel is marketed under brand names including Kymriah.

What are the contraindications for Tisagenlecleucel?

Tisagenlecleucel labeling lists contraindications including: None. None.. Always consult the full prescribing information and a clinician.