Legend Biotech Carvykti®, a Breakthrough CAR-T Cell Therapy
Legend Biotech Carvykti®, a Breakthrough CAR-T Cell Therapy
Latest news from Carvykti
Carvykti® (cilta-cel, Ciltacabtagene Autoleucel) is the first CAR-T cell therapy to receive a positive opinion from the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) for the second-line treatment of patients with relapsed or refractory multiple myeloma.
On February 23, 2024, Legend Biotech (NASDAQ: LEGN) announced in Somerset, New Jersey, USA, that the CHMP of the EMA recommended the approval of an extension of the therapeutic indication for Carvykti® (cilta-cel, Ciltacabtagene Autoleucel) to include adult patients with relapsed and refractory multiple myeloma who have received at least one prior line of therapy, including an immunomodulatory agent and a proteasome inhibitor, and have progressed on their last line of therapy and are refractory to lenalidomide. This Type II variation application was submitted to the EMA by Janssen-Cilag International N.V., an affiliate of Legend Biotech’s partner Janssen. The CHMP’s positive opinion will be reviewed by the European Commission, which will make the final approval decision.
Dr. Ying Huang, CEO of Legend Biotech, said, “This milestone underscores our commitment to bringing Carvykti® to more patients worldwide and earlier in their treatment journey. The CHMP’s positive opinion reflects our relentless efforts with Janssen to provide a new treatment option for patients and bring hope to them and their families.”
The CHMP’s recommendation for Carvykti® is supported by data from the CARTITUDE-4 study (NCT04181827), which is the first randomized Phase 3 study evaluating the efficacy and safety of Carvykti® compared to Pomalidomide, Bortezomib, and Dexamethasone (PVd) or Daratumumab, Pomalidomide, and Dexamethasone (DPd) in patients with relapsed and lenalidomide-refractory multiple myeloma after 1-3 prior lines of therapy.
Dr. Jesús San Miguel, Senior Consultant at the Clínica Universidad de Navarra, Professor of Medicine Hematology, and Head of the Clinical and Translational Myeloma Program, said, “The CARTITUDE-4 study results showed that patients with early relapse can derive significant clinical benefit from this therapy. The CHMP’s positive opinion is an important step towards bringing Carvykti®, a one-time infusion therapy, to more patients in Europe who can benefit from it.”
About Carvykti® (cilta-cel, Ciltacabtagene Autoleucel)
Ciltacabtagene Autoleucel is a B-cell maturation antigen (BCMA)-directed chimeric antigen receptor T-cell (CAR-T) therapy that uses a patient’s own T cells, which are genetically engineered with a CAR to recognize and eliminate cells expressing BCMA. BCMA is primarily expressed on the surface of malignant multiple myeloma B-lineage cells, mature B cells, and plasma cells. Ciltacabtagene Autoleucel’s CAR protein features two BCMA-targeting single-domain antibodies with high affinity for cells expressing BCMA, and upon binding to BCMA, the CAR promotes T-cell activation, proliferation, and subsequent target cell elimination.
In December 2017, Janssen entered into a worldwide collaboration and license agreement with Legend Biotech to develop and commercialize Ciltacabtagene Autoleucel. In February 2022, Ciltacabtagene Autoleucel was approved by the U.S. FDA, in May 2022 it received conditional marketing authorization from the European Commission (EC), and in September 2022 it was approved by the Japanese Ministry of Health, Labour and Welfare (MHLW) for the treatment of adult patients with relapsed or refractory multiple myeloma, under the trade name Carvykti®. Ciltacabtagene Autoleucel was granted Breakthrough Therapy Designation by the U.S. FDA in December 2019 and by the Chinese National Medical Products Administration (NMPA) in August 2020. Additionally, Ciltacabtagene Autoleucel received PRIority MEdicines (PRIME) designation from the European Commission in April 2019. Ciltacabtagene Autoleucel was granted Orphan Drug Designation by the U.S. FDA in February 2019, the European Medicines Agency (EMA) in February 2020, and the Japanese Ministry of Health, Labour and Welfare (MHLW) in June 2020. In March 2022, the EMA’s Committee for Orphan Medicinal Products unanimously recommended maintaining Orphan Designation for Ciltacabtagene Autoleucel based on clinical data (improved and sustained complete response rates after treatment).
About CARTITUDE-4
CARTITUDE-4 (NCT04181827) is an international, randomized, open-label Phase 3 study evaluating the efficacy and safety of Ciltacabtagene Autoleucel compared to Pomalidomide, Bortezomib, and Dexamethasone (PVd) or Daratumumab, Pomalidomide, and Dexamethasone (DPd) in adult patients with relapsed and lenalidomide-refractory multiple myeloma after 1-3 prior lines of therapy, with progression-free survival (PFS) as the primary endpoint.
About Multiple Myeloma
Multiple myeloma is an incurable blood cancer that starts in the bone marrow and is characterized by an excessive proliferation of plasma cells. It is estimated that in 2024, more than 35,000 people in the United States will be diagnosed with multiple myeloma, and more than 12,000 people will die from the disease. While some patients with multiple myeloma have no symptoms, others are diagnosed due to symptoms that can include bone problems, low blood counts, calcium elevation, kidney problems, or infections.
About Legend Biotech
Legend Biotech (NASDAQ: LEGN), established in 2014, is a global, clinical-stage biopharmaceutical company engaged in the discovery and development of novel cell therapies for oncology and other indications. Our team of over 1,800 employees across the United States, China, and Europe is dedicated to advancing breakthrough cell therapies to improve the lives of those in need. We are strategically guiding our lead product candidate, ciltacabtagene autoleucel (cilta-cel), an investigational BCMA-directed CAR-T cell therapy, through clinical development for the treatment of multiple myeloma. In 2022, cilta-cel was approved by the U.S. Food and Drug Administration, the Japanese Ministry of Health, Labour and Welfare, and received conditional marketing authorization from the European Commission under the trade name Carvykti®, making it the first BCMA CAR-T product approved for the treatment of patients with relapsed/refractory multiple myeloma. Legend Biotech has a comprehensive portfolio of additional autologous and allogenic CAR-T cell therapy product candidates for the treatment of hematological malignancies and solid tumors.