Legend Biotech new drug application approved in China

January 3, 2023 - 3 minutes
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Legend Biotech Corporation has announced that China’s National Medical Products Administration (NMPA) has formally accepted its new drug application (NDA) for ciltacabtagene autoleucel (cilta-cel).

This submission is based on data from the confirmatory phase 2 clinical study CARTIFAN-1, conducted in China, which evaluated the efficacy and safety of cilta-cel in adult patients with relapsed or refractory multiple myeloma who have received three or more prior lines of therapy, including a proteasome inhibitor and immunomodulatory drug.

Saijuan Chen, hematologist and molecular geneticist, academician of the Chinese Academy of Engineering, and principal investigator of the CARTIFAN-1 clinical trial, said: “Incidence and mortality rates of multiple myeloma have recently increased in China, and the disease remains incurable. As a result, there is a huge unmet medical need for new treatment options. The data from the CARTIFAN-1 study showed that cilta-cel provided deep and durable responses in patients with relapsed or refractory multiple myeloma. We hope this drug becomes available to eligible patients as soon as possible.”

Ying Huang, chief executive officer of Legend Biotech, said: “Meeting medical needs and serving patients around the world has always been the goal of Legend Biotech’s innovative research and development. Cilta-cel has been approved for marketing in the United States and Japan and has received conditional marketing authorization in Europe. We look forward to the possibility of providing a new treatment option for appropriate patients with relapsed and refractory multiple myeloma in China.”

About Ciltacabtagene autoleucel (cilta-cel)

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.

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

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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.

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