{"entity":{"id":"axicabtagene-ciloleucel","kind":"drug","name":"Axicabtagene ciloleucel","aka":[],"tldr":"A CD19 CAR-T that cures about 40% of patients with large B-cell lymphoma who had failed everything, and beat transplant in second line.","summary":"Axicabtagene ciloleucel (axi-cel) is an autologous CAR-T: a patient's T cells are engineered with a retroviral vector to express a CD19-binding scFv with a CD28 costimulatory domain and reinfused after cyclophosphamide and fludarabine lymphodepletion. It was approved in 2017 for large B-cell lymphoma after two or more lines (ZUMA-1; 5-year OS about 43%), in 2022 as second-line therapy for early relapse (ZUMA-7, OS benefit), and for follicular lymphoma. Kite/Gilead manufactures it. CD28 costimulation gives fast expansion and more neurotoxicity than 4-1BB products; cytokine release syndrome, prolonged cytopenias and infections are the other main risks. How to choose between CAR-T and CD20xCD3 bispecifics, and how to widen access, remain open. For a newcomer: a one-time living drug that cures a meaningful share of lymphomas with no standard treatment left to try.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Axicabtagene_ciloleucel","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Axicabtagene%20ciloleucel"},{"label":"Deng et al., Nat Med 2020: single-cell features of the axicabtagene ciloleucel infusion product that track efficacy and toxicity in 24 patients","url":"https://doi.org/10.1038/s41591-020-1061-7"},{"label":"Plaks et al., Blood 2021: CD19 target evasion as a mechanism of relapse after axicabtagene ciloleucel in large B-cell lymphoma","url":"https://doi.org/10.1182/blood.2021010930"},{"label":"Sotillo et al., Cancer Discov 2015: acquired mutations and alternative splicing of CD19 enable resistance to CART-19","url":"https://doi.org/10.1158/2159-8290.CD-15-1020"}],"tags":[],"related":[],"cancers":["dlbcl","primary-mediastinal-b-cell-lymphoma"],"sections":[],"technologies":["car-t"],"targets":["cd19"],"drugs":[],"companies":["gilead"],"institutions":[],"pathways":[],"terms":["lymphoma-bio-antigen-escape"],"trials":["nct05371093"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["Lymphoma biology: half the medicine is the patient's own T cells. Single-cell sequencing of 24 infusion products found three times the frequency of memory-signature CD8 T cells in patients who were in complete response at three months, and an exhaustion signature associated with a poor molecular response in cell-free DNA at day 7; a rare monocyte-like population in the product was associated with high-grade neurotoxicity (Deng 2020). The other failure mode is CD19 target evasion (Plaks 2021, Sotillo 2015)."],"brand":"Yescarta","code":"axi-cel","modality":"CAR-T (CD19)","mechanism":"CD19 scFv, CD28 costimulation, retroviral.","approvals":[{"region":"US","year":2017,"indication":"Relapsed/refractory large B-cell lymphoma ≥2 lines"},{"region":"US","year":2022,"indication":"Second-line LBCL refractory or relapsed within 12 months"}],"mechanismSteps":["Patient's T cells are collected by leukapheresis","Cells are engineered to express a CAR against CD19 and expanded","Patient receives lymphodepleting chemotherapy","CAR-T cells are infused, home to tumour, and expand","CAR binds CD19; T cell kills the tumour cell and proliferates","Memory CAR-T cells persist and patrol"],"dosing":{"route":"Single IV infusion after lymphodepletion","schedule":"2 × 10⁶ CAR+ T cells/kg (max 2 × 10⁸); cyclophosphamide 500 mg/m² + fludarabine 30 mg/m² days −5 to −3","modifications":"Tocilizumab ± steroids for CRS; steroids for ICANS; prophylactic steroids permitted","monitoring":"Daily monitoring for 7 days (outpatient permitted with rapid access), then twice weekly for 4 weeks; cytopenias, infections, B-cell aplasia","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},"toxicity":[{"event":"Cytokine release syndrome","note":"ZUMA-7: ~92% any grade, 6% grade ≥3"},{"event":"Neurologic toxicity","note":"ZUMA-7: ~60% any grade, 21% grade ≥3"},{"event":"Prolonged cytopenias"},{"event":"Infections"},{"event":"Hypogammaglobulinaemia"}],"access":[{"country":"US","listPrice":"$373,000 per infusion (list price at launch 2017; higher today)","reimbursement":"Medicare Part B (physician-administered); commercial plans per formulary","assistance":"https://www.kitekonnect.com","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NICE: recommended for relapsed/refractory DLBCL after ≥2 therapies (TA872) and second-line early relapse (TA895)","source":"https://www.nice.org.uk/guidance/published?ngt=Technology%20appraisal%20guidance","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2017-10-18","type":"approval","region":"US","note":"Relapsed/refractory large B-cell lymphoma after ≥2 lines (ZUMA-1): second CAR-T ever approved","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2021-03-05","type":"accelerated-approval","region":"US","note":"Relapsed/refractory follicular lymphoma (ZUMA-5) The confirmatory requirement was still open 5.5 years later, when the FDA's table was read.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Treatment of adult patients with relapsed or refractory follicular lymphoma (FL) after two or more lines of systemic therapy"},{"date":"2022-04-01","type":"approval","region":"US","note":"Second-line LBCL refractory or relapsed within 12 months (ZUMA-7)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2024-01-19","type":"label-change","region":"US","note":"Class boxed warning for T-cell malignancies","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},"route":"/drugs/axicabtagene-ciloleucel/","neighbours":{"cancer":[{"id":"burkitt-lymphoma","kind":"cancer","name":"Burkitt lymphoma","route":"/cancers/burkitt-lymphoma/"},{"id":"dlbcl","kind":"cancer","name":"Diffuse large B-cell lymphoma","route":"/cancers/dlbcl/"},{"id":"follicular-lymphoma","kind":"cancer","name":"Follicular lymphoma","route":"/cancers/follicular-lymphoma/"},{"id":"hiv-associated-lymphoma","kind":"cancer","name":"HIV-associated (AIDS-related) lymphomas","route":"/cancers/hiv-associated-lymphoma/"},{"id":"non-hodgkin-lymphoma","kind":"cancer","name":"Non-Hodgkin lymphoma (all types)","route":"/cancers/non-hodgkin-lymphoma/"},{"id":"post-transplant-lymphoproliferative-disorder","kind":"cancer","name":"Post-transplant lymphoproliferative disorder (PTLD)","route":"/cancers/post-transplant-lymphoproliferative-disorder/"},{"id":"primary-cns-lymphoma","kind":"cancer","name":"Primary CNS lymphoma","route":"/cancers/primary-cns-lymphoma/"},{"id":"primary-mediastinal-b-cell-lymphoma","kind":"cancer","name":"Primary mediastinal (thymic) large B-cell lymphoma","route":"/cancers/primary-mediastinal-b-cell-lymphoma/"}],"technology":[{"id":"car-t-manufacturing-process","kind":"technology","name":"Autologous CAR-T manufacturing, batch by batch","route":"/technologies/car-t-manufacturing-process/"},{"id":"car-t","kind":"technology","name":"CAR-T cell therapy","route":"/technologies/car-t/"}],"target":[{"id":"cd19","kind":"target","name":"CD19","route":"/targets/cd19/"},{"id":"cd28","kind":"target","name":"CD28","route":"/targets/cd28/"}],"company":[{"id":"gilead","kind":"company","name":"Gilead Sciences (incl. Kite)","route":"/companies/gilead/"},{"id":"lysa","kind":"company","name":"LYSA (The Lymphoma Study Association)","route":"/companies/lysa/"}],"term":[{"id":"lymphoma-bio-antigen-escape","kind":"term","name":"Antigen escape: how a lymphoma loses the thing the drug was aimed at","route":"/terms/lymphoma-bio-antigen-escape/"},{"id":"lymphoma-tx-crs-icans","kind":"term","name":"Cytokine release syndrome and ICANS: grading and management","route":"/terms/lymphoma-tx-crs-icans/"},{"id":"lymphoma-tx-immunoglobulin-replacement","kind":"term","name":"Immunoglobulin replacement after CAR-T, bispecifics and long anti-CD20 treatment","route":"/terms/lymphoma-tx-immunoglobulin-replacement/"},{"id":"lymphoma-type","kind":"term","name":"Lymphoma (tissue type)","route":"/terms/lymphoma-type/"},{"id":"lymphoma-tx-pod24","kind":"term","name":"POD24: progression of follicular lymphoma within two years, and why it changes the plan","route":"/terms/lymphoma-tx-pod24/"},{"id":"lymphoma-tx-car-t-pathway","kind":"term","name":"The CAR-T pathway in lymphoma: referral, apheresis, bridging and the waiting","route":"/terms/lymphoma-tx-car-t-pathway/"},{"id":"lymphoma-tx-uk-access","kind":"term","name":"What the NHS in England funds for lymphoma, appraisal by appraisal","route":"/terms/lymphoma-tx-uk-access/"}],"trial":[{"id":"nct06550141","kind":"trial","name":"Emapalumab Prevention of CAR-T Cell Associated Toxicities","route":"/trials/nct06550141/"},{"id":"nct05371093","kind":"trial","name":"Study of Axicabtagene Ciloleucel Versus Standard of Care Therapy in Participants With Relapsed/Refractory Follicular Lymphoma","route":"/trials/nct05371093/"},{"id":"zuma-1","kind":"trial","name":"ZUMA-1","route":"/trials/zuma-1/"},{"id":"zuma-5","kind":"trial","name":"ZUMA-5","route":"/trials/zuma-5/"},{"id":"zuma-7","kind":"trial","name":"ZUMA-7","route":"/trials/zuma-7/"}],"idea":[{"id":"idea-reg-cart-outcomes-refund","kind":"idea","name":"Full refund for CAR-T if the patient has not responded at three months","route":"/ideas/idea-reg-cart-outcomes-refund/"},{"id":"idea-bispecific-vs-car-t-second-line","kind":"idea","name":"Head-to-head bispecific vs CAR-T in second-line LBCL","route":"/ideas/idea-bispecific-vs-car-t-second-line/"},{"id":"idea-reg-vein-to-vein-public-benchmark","kind":"idea","name":"Mandatory public reporting of vein-to-vein time and failure rate per CAR-T product","route":"/ideas/idea-reg-vein-to-vein-public-benchmark/"},{"id":"lymphoma-ev-late-effects-of-the-treatments-given-now","kind":"idea","name":"Measure the late effects of the treatments being given now, not the ones given in 1975","route":"/ideas/lymphoma-ev-late-effects-of-the-treatments-given-now/"},{"id":"idea-fund-pay-for-cure-annuities","kind":"idea","name":"Outcome-based annuity payments for potentially curative one-time therapies","route":"/ideas/idea-fund-pay-for-cure-annuities/"},{"id":"idea-moon-pay-for-cure-contracts","kind":"idea","name":"Pay-for-cure contracts: instalment payments for curative therapies contingent on durable remission","route":"/ideas/idea-moon-pay-for-cure-contracts/"},{"id":"lymphoma-ev-manufacturing-time-as-a-trial-endpoint","kind":"idea","name":"Report the time from apheresis to infusion as a trial endpoint, not a logistics footnote","route":"/ideas/lymphoma-ev-manufacturing-time-as-a-trial-endpoint/"}],"paper":[{"id":"paper-zuma-5-axi-cel-indolent-lymphoma-lancet-oncol-2022","kind":"paper","name":"Axicabtagene ciloleucel in relapsed or refractory indolent non-Hodgkin lymphoma (ZUMA-5): a single-arm, multicentre, phase 2 trial","route":"/key-papers/paper-zuma-5-axi-cel-indolent-lymphoma-lancet-oncol-2022/"},{"id":"paper-zuma-1-axi-cel-nejm-2017","kind":"paper","name":"ZUMA-1: axicabtagene ciloleucel for refractory large B-cell lymphoma, the first CAR-T approved for lymphoma","route":"/key-papers/paper-zuma-1-axi-cel-nejm-2017/"},{"id":"paper-zuma-7-axi-cel-second-line-nejm-2022","kind":"paper","name":"ZUMA-7: axi-cel CAR-T instead of salvage chemotherapy and transplant for large B-cell lymphoma that relapses early","route":"/key-papers/paper-zuma-7-axi-cel-second-line-nejm-2022/"}],"roadmap":[{"id":"cell-therapy-roadmap","kind":"roadmap","name":"Cell therapy roadmap: CD19 CAR-T → solid tumours → in vivo CAR","route":"/roadmaps/cell-therapy-roadmap/"},{"id":"lymphoma-roadmap","kind":"roadmap","name":"Lymphoma roadmap: from a jaw tumour in Uganda and the first human cancer virus to gene-expression subtypes, PET-adapted chemotherapy, CAR-T cells, bispecific antibodies and the genetics-directed trials now recruiting","route":"/roadmaps/lymphoma-roadmap/"}],"person":[{"id":"frederick-locke","kind":"person","name":"Frederick L. Locke","route":"/people/frederick-locke/"},{"id":"jason-westin","kind":"person","name":"Jason R. Westin","route":"/people/jason-westin/"},{"id":"sattva-neelapu","kind":"person","name":"Sattva S. Neelapu","route":"/people/sattva-neelapu/"}],"biomarker":[{"id":"cd19-expression","kind":"biomarker","name":"CD19 expression (CD19-positive)","route":"/biomarkers/cd19-expression/"}]}}