China grants full approval to Tuoyi (toripalimab) for advanced urothelial carcinoma – Junshi Biosciences
Shanghai Junshi Biosciences Co., Ltd. announced that the National Medical Products Administration of China has converted from conditional approval to regular approval the toripalimab injection (trade name: TUOYI, product code: JS001) as monotherapy for the treatment of patients with locally advanced or metastatic urothelial carcinoma who failed platinum-containing chemotherapy, including those who progressed within 12 months of neoadjuvant or adjuvant platinum-containing chemotherapy.
UC is one of the ten most prevalent malignant tumors in the world, and in China, its incidence and mortality rates continue rising annually. It is a serious threat to the life and health of patients, and there are huge unmet clinical needs.
In April 2021, based on a multi-center, single-arm, open-label Phase II clinical trial (the POLARIS-03 study, NCT03113266), toripalimab obtained conditional marketing approval from the NMPA for the second-line and above treatment of advanced UC, becoming the first immunotherapeutic drug approved for non-selective population-based indications of advanced UC in China.
This regular approval is primarily based on results from the RC48-C016 study (NCT05302284). The study is a multi-center, randomized, open-label and active-controlled phase III clinical trial which was designed to compare the efficacy and safety of toripalimab in combination with disitamab vedotin versus gemcitabine in combination with cisplatin/carboplatin in patients who had not previously received systemic anti-tumor therapy with HER2 (human epidermal growth factor receptor 2)-expressing (defined as HER2 IHC 1+, 2+ or 3+) locally advanced or metastatic UC. The study was conducted in 74 clinical centers across China with Professor Guo Jun from Beijing Cancer Hospital and Professor Zhou Aiping from the Cancer Hospital of the Chinese Academy of Medical Sciences as the principal investigators.
In October 2025, the results of the RC48-C016 study were published on The New England Journal of Medicine (NEJM) and were simultaneously presented orally at the Presidential Symposium of the 2025 European Society for Medical Oncology (ESMO) Annual Meeting (abstract number: #LBA7). The results showed that the primary endpoints of progression-free survival (“PFS”, based on independent imaging assessment) and overall survival (“OS”) achieved “dual-endpoint positive results”. Compared with traditional chemotherapy, toripalimab in combination with disitamab vedotin as first-line treatment for HER2-expressing advanced urothelial carcinoma doubled the median PFS (13.1 months vs. 6.5 months, hazard ratio HR=0.36, 95% CI: 0.28-0.46; p<0.0001), significantly prolonged the median OS (31.5 months vs. 16.9 months, HR=0.54, 95% CI: 0.41-0.73; p<0.0001), substantially improved the objective response rate (ORR) (76.1% vs. 50.2%), and extended the median duration of response by several times (14.6 months vs. 5.6 months). The combination treatment group substantially improved safety compared with traditional chemotherapy.
To date, TUOYI has obtained marketing approval for 13 indications in Chinese Mainland, of which 12 indications have been included in the National Reimbursement Drug List (NRDL), and it is the only anti-PD-1 monoclonal antibody in the NRDL for the treatment of renal cancer, triple-negative breast cancer and melanoma.
