Baiji gene autologous anti-CLL-1 chimeric antigen receptor T cell injection was approved for clinical practice

It was learned from Baiji Gene's WeChat public account: On December 30, 2022, according to the official website of the Drug Inspection Center (CDE) of the State Food and Drug Administration, the clinical trial application of "Autologous Anti-CLL-1 Chimeric Antigen Receptor T Cell Injection" (acceptance number: CXSL2200512) of Guangzhou Baiji Gene Technology Co., Ltd. (hereinafter referred to as "Guangzhou Baiji") was approved. The indication is relapsed and refractory acute myeloid leukemia (r/r AML).

U.S. FDA orphan drug designation

In August 2021, the product received orphan drug designation (ODD) status from the U.S. Food and Drug Administration (FDA) for the treatment of acute myeloid leukemia (AML).

EU EMA orphan drug designation

In October 2022, it obtained orphan drug designation (ODD) from the European Medicines Agency (EMA) for the treatment of AML.

About anti-CLL-1 CAR-T products

Autologous anti-CLL-1 chimeric antigen receptor T cell injection is a cell therapy product independently developed by Guangzhou Baiji and has core patented technology targeting CLL1, which has the advantages of high specificity, high affinity, durable and efficient cancer cell killing ability of antibodies. In June 2021, Guangzhou Baiji orally reported data at the 2021 American Society of Clinical Oncology (ASCO) Annual Meeting showing that after infusion of CAR-T cells in 11 patients, CAR-T cells could be rapidly and efficiently expanded in children. The overall response rate (ORR) was as high as 81.8% (9/11) and the total disease control rate (DCR) was as high as 90.9% (10/11), and 10/11 patients responded positively to Anti-CLL1 CAR-T therapy within one month.

About ODD

In the United States, a rare disease is defined as a disease that affects fewer than 200,000 Americans; In the European Union, rare diseases are defined as diseases in which the proportion of patients is less than 5 in 10,000. Orphan drug designation is intended to develop new therapies for diseases that are low-morbid, life-threatening, or long-term debilitating to patients, and there must be sufficient nonclinical or clinical data to demonstrate better efficacy than existing therapies. The FDA and EMA provide a series of incentives for drugs that have obtained orphan drug designation, including market exclusivity, clinical plan assistance, centralized approval of marketing applications, and related fee reductions.