Milestone Achieved: CDE Accepts China's First CAR-T Therapy IND Application from Legend Biotech

December 2017Month11On [Date], Nanjing Legend Biotech Co., Ltd.(hereinafter referred to as Nanjing Legend)SubmittedCAR-TTherapy Clinical Trial Application in China (CXSL1700201) ObtainCDE(Center for Drug Evaluation)Officially accepted for handling.This is the domesticCAR-TThe first submission after the project was placed under drug regulatory oversightCAR-TClinical Trial Application for the Project.

Legend Biotech, established in 2014 as a wholly-owned subsidiary of GenScript, is dedicated to the research and development of chimeric antigen receptor (CAR) T-cell technology in immunotherapy. The company has historically maintained a low profile until June 5, 2017, when it presented clinical data on its BCMA-targeted CAR-T therapy at the ASCO Annual Meeting. The outstanding efficacy demonstrated by this CAR-T therapy in the treatment of multiple myeloma rapidly propelled it into the spotlight.

LCAR-B38M is a BCMA-targeted CAR-T therapy developed by Legend Biotech. Data from a clinical trial involving 35 patients with relapsed or refractory multiple myeloma showed that the therapy achieved an objective response rate (ORR) of 100%. Among the first 19 patients treated, Legend Biotech conducted follow-up observations for more than 4 to 14 months. Of these, 14 patients maintained stringent complete response (sCR), while 5 achieved partial response (PR).

Dr. Xiaohu (Frank) Fan, Chief Scientific Officer of Legend Biotech, revealed that the company plans to enroll 100 patients in clinical trials in China.

Nanjing Legend has submitted a registration application for its product as a Class 1 biological product (biological products not yet marketed domestically or internationally). The drug name is “LCAR-B38M CAR-T Cell Autologous Infusion Product (abbreviated as: LCAR-B38M Cell Product).”

The regulatory framework for CAR-T therapy in China has become clearer, with such therapies to be regulated as drugs. They must first obtain clinical trial approval from the CFDA and, upon completion of clinical trials, submit an application for market authorization.

Although there is still a considerable distance from “document acceptance” to “document approval,” and the journey from the initiation of clinical trials to product commercialization is even longer, the CDE’s acceptance of the application materials marks the first formal step for CAR-T therapy in China. This milestone event holds significant importance and will provide substantial encouragement to the hundreds of domestic CAR-T therapy companies.