Guangzhou Bioheal Gene Technology's Anti-CLL1 CAR-T Therapy Receives IND Approval in China for Relapsed/Refractory Acute Myeloid Leukemia

According to the WeChat Official Account of Guangzhou Bio-gene Technology Co., Ltd.: On December 30, 2022, the official website of the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) showed that,Guangzhou Bio-gene Technology Co., Ltd. ("Guangzhou Bio-gene" for short) has received approval for the clinical trial application (acceptance number: CXSL2200512) of its "Autologous Anti-CLL-1 Chimeric Antigen Receptor T-Cell Injection." The indication is relapsed or refractory acute myeloid leukemia (r/r AML).

FDA Orphan Drug Designation in the United States

In August 2021, the product was granted Orphan Drug Designation (ODD) by 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 received orphan drug designation (ODD) from the European Medicines Agency (EMA) for the treatment of AML.

About Anti-CLL-1 CAR-T Product

The autologous anti-CLL-1 chimeric antigen receptor T-cell injection, developed by Guangzhou Bio-gene Technology Co., Ltd with independent core patented technology targeting CLL1, is a cellular therapy product. It features high specificity and high affinity of the antibody, as well as persistent and highly efficient cancer cell-killing capabilities. In June 2021, Guangzhou Bio-gene Technology Co., Ltd presented oral report data at the 2021 American Society of Clinical Oncology (ASCO) Annual Meeting showing that after infusing CAR-T cells into 11 patients, the CAR-T cells expanded rapidly and efficiently within the patients' bodies; the overall response rate (ORR) reached 81.8% (9/11), and the overall disease control rate (DCR) was as high as 90.9% (10/11). Ten out of eleven patients showed a positive response to the Anti-CLL1 CAR-T therapy within one month.

About ODD

In the United States, a rare disease is defined as a condition that affects fewer than 200,000 Americans; in the European Union, a rare disease is defined as one with a prevalence of less than 5 per 10,000 people. Orphan drug designation aims to develop new therapies for diseases with low incidence rates that are life-threatening or cause long-term debilitation, and there must be sufficient non-clinical or clinical data to demonstrate that the drug offers better efficacy than existing treatments. The FDA and EMA provide a series of incentives for drugs granted orphan drug designation, including market exclusivity, assistance with clinical development plans, centralized review of marketing applications, and fee reductions.

Editor: Dada Xiwa

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