Cadonilimab (PD-1/CTLA-4 Bispecific Antibody) Obtained Orphan Drug Designation from FDA of the United States for Treating Cervical Cancer

On February 23, 2021, Akeso, Inc. (9926.HK) announced that, the first-in-class novel drug Cadonilimab (PD-1/CTLA-4 bi-specific antibody, research and development code: AK104), which is a novel immuno-oncology therapy independently developed by the Company, has obtained orphan drug designation from the Food and Drug Administration of the United States (“FDA”) for treating cervical cancer (except very early stage IA1).


This represents another significant progress after obtaining fast track designation (FTD) from the FDA and the “Breakthrough Therapy Designation” from the National Medical Products Administration (NMPA) of the People’s Republic of China (the “PRC”) in 2020 for Cadonilimab treating patients with recurrent or metastatic cervical cancer after standard therapies.


Currently, there are no approved standard treatment for cervical cancer patient who had received a failed platinum-based chemotherapy. The objective response rate (ORR) for laterline treatment is less than 10%, the progression-free survival is short, the chemotherapy dosage tolerance is low, and the incidence of adverse reactions is relatively high. There is an urgent demand for effective drugs to improve patient treatment. The enrollment of patient in registrational Phase II clinical trial of Cadonilimab for treating patients with recurrent or metastatic cervical cancer after standard therapies has been completed in the PRC.  


Originated from the Orphan Drug Act of 1983, drug candidates with orphan drug designation have the opportunity to gain seven years of market exclusivity, along with a series of comprehensive benefits provided by the FDA, including tax credits, exemption from biologics license application fees, deduction of or exemption from prescription drug user fees, research and development funding support, protocol assistance, and accelerated regulatory approval.






Cadonilimab (AK104) is a novel, potential next-generation, first-in-class bi-specific PD-1/ CTLA-4 immunooncology backbone drug independently developed by the Company, and its major indications include liver cancer, cervical cancer, lung cancer, gastric cancer, esophageal squamous cell cancer and nasopharyngeal carcinoma. The preliminary research data of cervical cancer, gastric cancer and other tumors shows that, as compared with the combination therapy of PD-1 and CTLA-4, Cadonilimab has much lower toxicity and demonstrated promising safety profile and efficacy. Our AK104 project has been incorporated in the Major New Drug Innovation Program under the 13th Five-year Plan for Major Technology Project (十三五「重大新藥創製」科技重大專項支持專案) issued by National Health Commission and Ministry of Science and Technology in 2017 and has been enlisted in the 2017 Pearl River Talent Program of Guangdong Province — Introduction of Innovation and Entrepreneurship Team Support Program (2017年廣東 省「珠江人才計劃」引進創新創業團隊支持專案). It was also jointly rated by China Medical Biotechnology Association and Chinese Medicinal Biotechnology as one of the 2017 Top Ten Medicinal Biotechnology Advancements in China (2017年中國醫藥生物技 術十大進展).




Akeso, Inc. (9926.HK) is a biopharmaceutical company dedicated to the research, development, manufacturing and commercialization of new innovative antibody drugs that are affordable to patients worldwide. Since the Company’s establishment, the Company has established an end-to-end comprehensive drug development platform (ACE Platform) and system, encompassing fully integrated drug discovery and development functions, including target validation, antibody drug discovery and development, CMC production process development, and GMP compliant scale production. The Company has also successfully developed a bi-specific antibody drug development technology (Tetrabody technology). The Company currently has a pipeline of over 20 innovative drugs for the treatment of major diseases like tumors, autoimmune diseases, inflammation and metabolism diseases, 13 of which have entered clinical stage, including two first-in-class bi-specific antibody drugs (PD-1/CTLA-4 and PD-1/VEGF). The Company’s vision is to become a global leading biopharmaceutical company through research and development of high efficacy and breakthrough new drugs that are first-in-class and best-in-class therapies.