New indication for this one-time infusion may provide patients with a treatment-free respite as early as first relapse

SOMERSET, NJ, USA I April 05, 2024 I Legend Biotech Corporation (NASDAQ: LEGN) (Legend Biotech), a global leader in cell therapy, announced today the U.S. Food and Drug Administration (FDA) has approved CARVYKTI® (ciltacabtagene autoleucel; cilta-cel) for the treatment of patients with relapsed or refractory multiple myeloma who have received at least one prior line of therapy, including a proteasome inhibitor (PI), and an immunomodulatory agent (IMiD), and are refractory to lenalidomide.1 CARVYKTI® is the first and only B-cell Maturation Antigen (BCMA) targeted therapy, including CAR-T therapies, bispecific antibodies, and antibody-drug conjugates (ADCs), approved starting in the second-line of treatment for patients with multiple myeloma.

“The expanded label of CARVYKTI has the potential to transform the treatment paradigm for multiple myeloma by providing patients and physicians with a personalized immunotherapy that can be used earlier in the treatment regimen. Multiple myeloma is an incurable and progressive hematologic cancer that causes patients to relapse and become refractory to treatment, so new, innovative options are desperately needed,” said Ying Huang, Ph.D., Chief Executive Officer of Legend Biotech. “We are committed to improving the lives of patients battling blood cancer and will continue to work towards developing cellular therapies that bring us closer to a cure.”

The FDA approval is based on positive results from the CARTITUDE-4 study, which demonstrated that CARVYKTI® resulted in statistically significant and clinically meaningful improvement of progression-free survival compared to two standard of care treatment regimens, pomalidomide, bortezomib, and dexamethasone (PVd) or daratumumab, pomalidomide, and dexamethasone (DPd), in adults with relapsed and lenalidomide-refractory multiple myeloma who received one to three prior lines of therapy.1 This approval follows a unanimous (11 to 0) recommendation from the FDA Oncologic Drugs Advisory Committee (ODAC) in support of CARVYKTI® in earlier lines of treatment.

“The results of the CARTITUDE-4 study demonstrated the substantial clinical benefit of cilta-cel infusion over standard of care continuous therapy in patients who experience a relapse after one to three prior treatments,” said Surbhi Sidana, M.D., Assistant Professor of Medicine, Blood and Marrow Transplantation & Cellular Therapy at Stanford University School of Medicine.† “This expanded FDA approval for cilta-cel will allow a wider patient population to access this novel therapy earlier in the course of their treatment.”

“This expanded FDA approval validates the growing need for CARVYKTI and means more patients will have access to our life-altering, individualized therapy,” said Birk Vanderweeën, Senior Vice President, Global Manufacturing and Supply. “To ensure we can meet increased demand for our customized therapy, our global manufacturing teams and partners have expanded production capacity and are laser-focused on further scaling out operations to ensure patients who need CARVYKTI have access to it.”

The safety profile for CARVYKTI® includes a boxed warning for Cytokine Release Syndrome (CRS), Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS), Parkinsonism and Guillain-Barre syndrome, and their associated complications, Hemophagocytic Lymphohistiocytosis/Macrophage Activation Syndrome (HLH/MAS), Prolonged and Recurrent Cytopenias and Secondary Malignancies including myelodysplastic syndrome, acute myeloid leukemia, and T-cell malignancies.1 Warnings and Precautions include Increased Early Mortality, Infections, Hypogammaglobulinemia, Hypersensitivity Reactions, and Effects on Ability to Drive and Use Machines.1

The 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.1 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.1

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. 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.1

In December 2017, Legend Biotech entered into an exclusive worldwide license and collaboration agreement with Janssen Biotech, Inc. (Janssen), a Johnson & Johnson company, to develop and commercialize cilta-cel. 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. In May 2022, the European Commission (EC) granted conditional marketing authorization of CARVYKTI® for the treatment of adults with relapsed and refractory multiple myeloma. In September 2022, Japan’s Ministry of Health, Labour and Welfare (MHLW) approved CARVYKTI®. 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.


CARTITUDE-4 (NCT04181827) is an ongoing, international, randomized, open-label Phase 3 study evaluating the efficacy and safety of cilta-cel versus pomalidomide, bortezomib and dexamethasone (PVd) or daratumumab, pomalidomide, and dexamethasone (DPd) in adult patients with relapsed and lenalidomide-refractory multiple myeloma who received one to three prior lines of therapy, including a PI and an IMiD. The primary endpoint of the study was progression-free survival.2


Multiple myeloma is an incurable blood cancer that starts in the bone marrow and is characterized by an excessive proliferation of plasma cells.3 In 2024, it is estimated that more than 35,000 people will be diagnosed with multiple myeloma, and more than 12,000 people will die from the disease in the U.S.4 While some patients with multiple myeloma initially have no symptoms, most patients are diagnosed due to symptoms that can include bone problems, low blood counts, calcium elevation, kidney problems or infections.5


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 (gd 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.

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†Surbhi Sidana, M.D., Assistant Professor of Medicine, Blood and Marrow Transplantation & Cellular Therapy at Stanford University School of Medicine has provided consulting and advisory services to Legend Biotech; she has not been paid for any media work.


1 CARVYKTI Prescribing Information. Horsham, PA: Janssen Biotech, Inc.
2 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). Accessed March 2024.
3 American Cancer Society. ”What is Multiple Myeloma?”. Available at: Accessed March 2024.
4 American Cancer Society. “Key Statistics About Multiple Myeloma.” Available at: Accessed March 2024.
5 American Cancer Society. Multiple myeloma: early detection, diagnosis, and staging. Available at: Accessed March 2023.

SOURCE: Legend Biotech