PNS127 THE DEVELOPMENT OF CHIMERIC ANTIGEN RECEPTOR (CAR-T) CELL THERAPY IN CHINA

Abstract

China has become one of the most active players in chimeric antigen receptor (CAR)-T cell therapy development with the largest number of clinical trials currently being conducted in China. This study aims to describe the current CAR-T cell therapy regulatory environment and development status in China. Chinese drug regulatory authority official websites: China Food and Drug Administration (CFDA) and Center for Drug evaluation (CDE) were consulted to identify relevant regulatory policies and CAR-Ts currently under development. CAR-T cell therapy was regulated as a medical technique subject to less stringent evidence requirements before “Guideline on the research and review process of cellular products” was released in 18th December, 2017. Not only clarifications on the CAR-T specific regulatory requirements were provided in this guideline, but also a more flexible approach for evidence assessments was introduced, such as the acceptance of non-registered clinical data for market authorization application (MAA). Other regulatory incentives included 12-year data protection for innovative biopharmaceuticals, allowance of overseas clinical trial data for MAA, and the implementation of priority review and approval pathway for innovative drugs with unmet clinical needs. Since the approval of first CAR-T cell therapy clinical trial in December 2017, 16 applications (12 CD19-targeted, 3 BCMA-targeted, and 1 GPC-3-targeted) for CAR-T cell therapies clinical trials have been accepted by China CDE to date. A trend of worldwide collaborations between international pharmaceutical giants and China biotech companies has been witnessed, such as the establishment of cooperative business between Janssen and Nanjing Legend Biotech. A range of regulations and incentives were newly adopted in order to create a supportive environment for innovative therapies in China. Thanks to these regulations, an important number of promising CAR-T cell therapies targeting life-threatening diseases is expected to reach the Chinese market in the upcoming years.