Update on Priority Review by CDE: Acceptance of First CAR-T Therapy Clinical Application

On December 11, 2017, the CAR-T therapy clinical application in China (CXSL1700201) filed by Nanjing Legend Biotechnology Co., Ltd. (hereinafter referred to as Nanjing Legend) was formally undertaken and accepted by the CDE (Center for Drug Evaluation, CFDA), which is the first CAR-T project clinical application after China regulates CAR-T projects as drug.

Established in 2014, Nanjing Legend is a wholly-owned subsidiary of Genscript and concentrates on R&D of the chimeric antigen receptor cell technology in the immunotherapy. This company has been low-key until June 5, 2017 when the company released the clinical data of the CAR-T therapy targeting BCMA in ASCO meeting, with the excellent results of its CAR-T therapy for treatment of multiple myeloma rapidly putting it under the spotlight.

LCAR-B38M is a CAR-T therapy developed by Nanjing Legend that targets BCMA, which had 100% overall response rate in a clinical trial with participation of 35 patients with relapsed or drug resistance multiple myeloma, according to data. Nanjing Legend observed and followed up the 19 patients who were earliest in receiving the treatment for 4-14 months, wherein, 14 patients continued to reach the stringent complete response diagnostic criteria and 5 showed partial response.

According to Dr. Xiaohu (Frank) Fan, the Chief Science Officer of Nanjing Legend, the company plans to recruit 100 patients in China to participate in the clinical trial.

Nanjing Legend filed the registration application according to the Class 1 biological product (biological products not marketed in China or overseas), with the pharmaceutical product named "LCAR-B38M CAR T-Cell Autotransfusion Preparation (LCAR-B38M Cell Preparation for short) ".

The regulatory idea of CAR-T therapies becomes clearer in China: they will be managed as pharmaceutical product, and shall first receive CFDA’s clinical approval, and be applied for marketing after completion of clinical trial.

There is still some way from "document acceptance" to "document approval", and it takes even a long way to go from clinical trial beginning to product marketing, however, CDE’s acceptance of the materials means that the CAR-T therapy has formally taken the first step in China, which is of milestone meaning, and will greatly encourage the hundreds of CAR-T therapy companies in China.

Read more: Update on Priority Review by CDE: Biggest Bright Spot Being the Inclusion of Novartis’ LCZ696

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