Legend Biotech Announces Submission to the European Medicines Agency for Expanded Use of CARVYKTI® (ciltacabtagene autoleucel)

The application to the European Medicines Agency is supported by data from the Phase 3 CARTITUDE-4 study, evaluating the safety and efficacy of cilta-cel in the treatment of patients with relapsed and lenalidomide-refractory multiple myeloma who received one to three prior lines of therapy

SOMERSET, NJ, USA I May 25, 2023 I Legend Biotech Corporation (NASDAQ: LEGN) (Legend Biotech), a global biotechnology company developing, manufacturing and commercializing novel therapies to treat life-threatening diseases, announced today that a Type II variation application was submitted to the European Medicines Agency (EMA) for CARVYKTI® based on data from the CARTITUDE-4 study (NCT04181827), which investigates the treatment of adult patients with relapsed and lenalidomide-refractory multiple myeloma who have received one to three prior lines of therapy. The submission was filed to the EMA by Janssen-Cilag International N.V., an affiliate of Janssen Biotech, Inc., Legend Biotech’s collaborator for the development and commercialization of cilta-cel.

“We are committed to investigating the full potential of CARVYKTI® across patient populations, including in earlier lines of treatment,” said Ying Huang, Ph.D., CEO of Legend Biotech. “I am proud of the efforts that Legend Biotech and our collaborator, Janssen, continue to make to potentially bring CARVYKTI® to a broader range of patients.”

The application is supported by data from the CARTITUDE-4 study, a randomized Phase 3 study evaluating the efficacy and safety of cilta-cel versus pomalidomide, bortezomib and dexamethasone (PVd) or daratumumab, pomalidomide and dexamethasone (DPd) in the treatment of patients with relapsed and lenalidomide-refractory multiple myeloma who received one to three prior lines of therapy.1 CARTITUDE-4 is the first randomized Phase 3 study investigating the efficacy of a cell therapy as early as after first relapse in multiple myeloma.1,2

Results from the CARTITUDE-4 study will be presented in an oral presentation session at the upcoming American Society of Clinical Oncology (ASCO) Annual meeting (Abstract #LBA106) on Monday, June 5, 2023 at 9:45 am – 11:15 am CDT. In addition, these data will also be presented at the 2023 European Hematology Association (EHA) Hybrid Congress as an oral presentation in a plenary session (Abstract #S100). A regulatory filing to the U.S. Food and Drug Administration is also planned.


CARVYKTI® (ciltacabtagene autoleucel) 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, after four or more prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody.

Please read full Prescribing Information including Boxed Warning for CARVYKTI®.


Ciltacabtagene autoleucel is a BCMA-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) that identifies and eliminates cells that express BCMA. BCMA is primarily expressed on the surface of malignant multiple myeloma B-lineage cells, as well as late-stage B-cells and plasma cells. The cilta-cel CAR protein features two BCMA-targeting single domain antibodies designed to confer high avidity against human BCMA. Upon binding to BCMA-expressing cells, the CAR promotes T-cell activation, expansion, and elimination of target cells.3

In December 2017, Legend Biotech entered into an exclusive worldwide license and collaboration agreement with Janssen Biotech, Inc. (Janssen) to develop and commercialize cilta-cel.

In May 2022, the European Commission (EC) granted conditional marketing authorization of CARVYKTI® for the treatment of adults with relapsed and refractory multiple myeloma.4,5 In February 2022, cilta-cel was approved by the U.S. Food and Drug Administration (FDA) under the brand name CARVYKTI® for the treatment of adults with relapsed or refractory multiple myeloma.6 In September 2022, Japan’s Ministry of Health, Labour and Welfare (MHLW) approved CARVYKTI®.7 Cilta-cel was granted Breakthrough Therapy Designation in the U.S. in December 2019 and in China in August 2020. In addition, cilta-cel received a PRIority MEdicines (PRIME) designation from the European Commission in April 2019. Cilta-cel also received Orphan Drug Designation from the U.S. FDA in February 2019, from the European Commission in February 2020, and from the Pharmaceuticals and Medicinal Devices Agency (PMDA) in Japan in June 2020. In March 2022, the European Medicines Agency’s Committee for Orphan Medicinal Products recommended by consensus that the orphan designation for cilta-cel be maintained on the basis of clinical data demonstrating improved and sustained complete response rates following treatment.8


Multiple myeloma is an incurable blood cancer that starts in the bone marrow and is characterized by an excessive proliferation of plasma cells.9 In Europe, more than 50,900 people were diagnosed with multiple myeloma in 2020, and more than 32,500 patients died.10 While some patients with multiple myeloma have no symptoms at all, most patients are diagnosed due to symptoms that can include bone problems, low blood counts, calcium elevation, kidney problems or infections.11 Although treatment may result in remission, unfortunately, patients will most likely relapse.12 Patients who relapse after treatment with standard therapies, including protease inhibitors, immunomodulatory agents, and an anti-CD38 monoclonal antibody, have poor prognoses and few treatment options available.13,14


Legend Biotech is a global biotechnology company dedicated to treating, and one day curing, life-threatening diseases. Headquartered in Somerset, New Jersey, we are developing advanced cell therapies across a diverse array of technology platforms, including autologous and allogeneic chimeric antigen receptor T-cell, gamma-delta T cell (γδ T) and natural killer (NK) cell-based immunotherapy. From our three R&D sites around the world, we apply these innovative technologies to pursue the discovery of cutting-edge therapeutics for patients worldwide.

Learn more at www.legendbiotech.com and follow us on Twitter and LinkedIn.


1 ClinicalTrials.Gov: 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 Myeloma (CARTITUDE-4). Available at: https://clinicaltrials.gov/ct2/show/NCT04181827?term=JNJ-68284528&phase=2&draw=2&rank=1. Accessed May 2023.
2 ClinicalTrials.gov. Search results for CAR-T, Multiple Myeloma, Phase 3 studies. Available at: https://clinicaltrials.gov/ct2/results?term=CAR-T&cond=Multiple+Myeloma&age_v=&gndr=&type=&rslt=&phase=2&Search=Apply. Last accessed: May 2023.
3 CARVYKTI™ Prescribing Information. Horsham, PA: Janssen Biotech, Inc.
4 CARVYKTI (ciltacabtagene autoleucel) Granted Conditional Approval by the European Commission for the Treatment of Patients with Relapsed and Refractory Multiple Myeloma. Available at: https://legendbiotech.com/legend-news/carvykti-ciltacabtagene-autoleucel-granted-conditional-approval-by-the-european-commission-for-the-treatment-of-patients-with-relapsed-and-refractory-multiple-myeloma/. Accessed March 2023.
5 European Medicines Agency. CARVYKTI (Ciltacabtagene Autoleucel) Summary of Product Characteristics. Available at: https://www.ema.europa.eu/en/documents/product-information/carvykti-epar-product-information_en.pdf. Last accessed: May 2023.
6 CARVYKTI™ (ciltacabtagene autoleucel), BCMA-Directed CAR-T Therapy, Receives U.S. FDA Approval for the Treatment of Adult Patients with Relapsed or Refractory Multiple Myeloma. Available at: https://legendbiotech.com/legend-news/carvykti-ciltacabtagene-autoleucel-bcma-directed-car-t-therapy-receives-u-s-fda-approval-for-the-treatment-of-adult-patients-with-relapsed-or-refractory-multiple-myeloma/. Accessed March 2023.
7 CARVYKTI™ (ciltacabtagene autoleucel) Receives Approval from Japan’s Ministry of Health, Labour and Welfare (MHLW) for the Treatment of Patients with Relapsed or Refractory Multiple Myeloma. Available at: https://www.businesswire.com/news/home/20220926005847/en/CARVYKTI%E2%84%A2-ciltacabtagene-autoleucel-Receives-Approval-from-Japan%E2%80%99s-Ministry-of-Health-Labour-and-Welfare-MHLW-for-the-Treatment-of-Patients-with-Relapsed-or-Refractory-Multiple-Myeloma. Accessed March 2023.
8 European Commission. Community Register of Orphan Medicinal Products. Available at: https://ec.europa.eu/health/documents/community-register/html/o2252.htm. Accessed March 2023.
9 American Society of Clinical Oncology. Multiple myeloma: introduction. https://www.cancer.net/cancer-types/multiple-myeloma/introduction. Accessed March 2023.
10 GLOBOCAN 2020. Cancer Today Population Factsheets: Europe Region. Available at: https://gco.iarc.fr/today/data/factsheets/populations/908-europe-fact-sheets.pdf Last accessed: April 2023.
11 American Cancer Society. Multiple myeloma: early detection, diagnosis and staging. https://www.cancer.org/content/dam/CRC/PDF/Public/8740.00.pdf. Accessed March 2023.
12 Rajkumar SV. Multiple myeloma: 2020 update on diagnosis, risk-stratification and management. Am J Hematol. 2020;95(5),548-567. doi:10.1002/ajh.25791.
13 Kumar SK, Dimopoulos MA, Kastritis E, et al. Natural history of relapsed myeloma, refractory to immunomodulatory drugs and proteasome inhibitors: a multicenter IMWG study. Leukemia. 2017;31(11):2443- 2448.
14 Gandhi UH, Cornell RF, Lakshman A, et al. Outcomes of patients with multiple myeloma refractory to CD38- targeted monoclonal antibody therapy. Leukemia. 2019;33(9):2266-2275.

SOURCE: Legend Biotech

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