Legend Biologics submitted a new drug marketing application for cedarchicel and was accepted by the China Food and Drug Administration

On January 2, 2023, Legend Bio's WeChat public account posted that the company's first self-developed cell therapy product, Ciltacabtagene Autoleucel (research code LCAR-B38M cell preparation), has been officially accepted by the National Medical Products Administration (NMPA).

Cedaki Cel-cel injection is an autologous T cell product (CAR-T cell) that targets B-cell mature antigen (BCMA) modified by chimeric antigen receptor gene, and is administered as a one-time infusion. Cedakicel received the first clinical trial application (IND) approval for CAR-T cell products from the National Medical Products Administration in March 2018, and became the first variety to be included in the breakthrough therapy program in China in August 2020.

This submission is based on the results of the confirmatory clinical study CARTIFAN-1 (NCT03758417/CTR20181007) conducted in China, which confirms the clinical benefit of cedarchicel in patients with relapsed or refractory multiple myeloma. This study evaluated the efficacy and safety of cedarchi-cel in adults with relapsed or refractory multiple myeloma (R/R MM) who had received at least 3 lines of prior therapy, including a proteasome inhibitor (PI) and an immunomodulator (IMiD).

Chen Saijuan, a Chinese hematologist and molecular geneticist, academician of the Chinese Academy of Engineering, and principal investigator of the CARTIFAN-1 clinical trial, said:

Multiple myeloma is the second most common hematologic malignancy in China, with a high incidence in middle-aged and elderly people. According to statistics, the age-standardized incidence of multiple myeloma in China in 2020 was about 1.15 per 100,000 people1. As China enters an aging society, the incidence rate has increased significantly, and the age of onset tends to be younger. Although survival has improved significantly in recent years, many patients are diagnosed at an advanced stage, most relapse after initial treatment, and have a poor prognosis. According to the data of CARTIFAN-1 confirmatory clinical trial conducted in China, cedarchicel has shown lasting and deep benefit in patients with relapsed or refractory multiple myeloma. We look forward to the early approval of this new CAR-T cell therapy drug to benefit more patients."

Dr. Ying Huang, CEO of Legendary Biologics, said:

Meeting clinical needs and serving patients around the world, including Chinese patients, has always been the original intention of Legend Bio's innovative research and development. Currently, Cedarkecel has been approved for marketing in the United States, Europe and Japan. As an original innovative drug born in China, it is of special significance that the marketing application of cedarcel has been officially accepted by the State Food and Drug Administration. We look forward to the early approval of this product in China, providing new treatment options for multiple myeloma patients in China and bringing more hope for cure."

About Ciltacabtagene Autoleucel

Cedaki Orencel is a chimeric antigen receptor T cell (CAR-T) product targeting B-cell maturation antigen (BCMA) that uses a transgene of chimeric antigen receptor (CAR) to modify a patient's own T cells to recognize and eliminate BCMA-expressing cells. BCMA is mainly expressed on the surface of malignant multiple myeloma B cells, advanced B cells and plasma cells. The CAR protein of cedarcel has two BCMA-targeted single-domain antibodies and has a high affinity for BCMA-expressing cells, and after binding to BCMA-expressing cells, CAR can promote T cell activation, expansion, and then elimination of target cells.

In December 2017, Janssen signed an exclusive worldwide licensing and cooperation agreement with Legendary Biologics to develop and commercialize Cedarchi Celest. In August 2020, Cedakicel received China's first "breakthrough therapy drug" designation, in April 2019 it received the European Commission's Priority Drug Designation (PRIME), and in December 2019 it was granted breakthrough therapy designation by the US FDA. The U.S. FDA, EMA and PMDA of Japan granted orphan drug designation to cedarchicel in February 2019, February 2020 and June 2020, respectively. In February 2022, Cedakicel received approval from the US Food and Drug Administration (FDA) under the trade name CARVYKTI®, in May it received conditional marketing authorization from the European Commission (EC), and in September it was approved by the Japanese Ministry of Health, Labour and Welfare (MHLW) for the treatment of relapsed or refractory multiple myeloma in adults.

About CARTIFAN-1

CARTIFAN-1 (NCT03758417) is a Phase 2 open-label, confirmatory trial to evaluate the efficacy and safety of cedarchicel in patients with relapsed or refractory multiple myeloma (R/R MM) in China who have received at least 3 lines of prior therapy, including proteasome inhibitors and immunomodulators, with the primary endpoint of overall response rate.

About multiple myeloma

Multiple Myeloma (MM) is considered an incurable hematological tumor and is a malignant disease caused by excessive proliferation of plasma cells in the bone marrow. According to the latest data from the World Health Organization, there were more than 21,000 new cases and nearly 16,200 deaths due to multiple myeloma in China in 20202222. Although some people with multiple myeloma have no obvious symptoms, most are diagnosed due to the presence of symptoms, which may include bone disease, abnormal low blood counts, elevated blood calcium, kidney problems, or infections. While there may be some remission with treatment, unfortunately, patients are likely to relapse. Patients who relapse after treatment with standard therapies, including protease inhibitors, immunomodulators, and anti-CD38 monoclonal antibodies, face a poor prognosis and limited treatment.