TransThera Sciences (Nanjing), Inc. (HKG: 2617) announced that the first nationwide prescription for its independently developed Class 1 innovative drug YOCHANRA® (tinengotinib tablets) was written on 17 Sep 2026 at Peking University Cancer Hospital, where Professor Zhou Jun of the Department of Gastrointestinal Oncology prescribed the drug to a patient with FGFR2 fusion/rearrangement advanced cholangiocarcinoma previously treated with systemic therapy and an FGFR inhibitor. The milestone marks the formal entry into clinical practice of the world’s first approved targeted therapy for cholangiocarcinoma progressing after FGFR inhibitor treatment, giving drug‑resistant patients in China a newly accessible treatment option.
Regulatory Milestone
| Item | Detail |
|---|---|
| Agency | NMPA (China), via priority review and conditional approval pathway |
| Approval Date | 6 Aug 2026 |
| Product | YOCHANRA® (tinengotinib tablets), Class 1 innovative drug |
| Indication | Adults with advanced, metastatic, or unresectable cholangiocarcinoma harboring FGFR2 fusion or rearrangement, previously treated with systemic therapy and an FGFR inhibitor |
| First Prescription | 17 Sep 2026, Peking University Cancer Hospital (Prof. Zhou Jun, FIRST‑08 principal investigator) |
| Prior Designations | NMPA Priority Review and Breakthrough Therapy Designation; US FDA Fast Track and Orphan Drug designations (cholangiocarcinoma); EMA Orphan Drug designation (biliary tract cancer); selected into China’s “13th Five‑Year” National Major Sci‑Tech Program for New Drug Creation |
Drug Profile & Mechanism of Action
- Molecule: Tinengotinib, an independently developed innovative multi‑target small‑molecule kinase inhibitor covering FGFR, VEGFR, JAK, and Aurora kinase targets.
- Triple Mechanism: Exerts antitumor activity through precision targeting, lineage remodeling, and immune activation.
- Resistance‑Breaking Design: A unique FGFR binding mode enables tinengotinib to overcome multi‑site resistance mutations while maintaining high binding activity – the structural basis for its approval in the post‑FGFR‑inhibitor setting.
- First‑in‑Class Position: The world’s first approved targeted drug for FGFR2 fusion/rearrangement cholangiocarcinoma after progression on prior FGFR inhibitor therapy.
Clinical Evidence – FIRST‑08 Pivotal Phase II Trial
| Endpoint (BICR) | Result |
|---|---|
| Objective Response Rate (ORR) | 28.0 % |
| Disease Control Rate (DCR) | 82.0 % |
| Median Duration of Response (DoR) | 8.5 months |
| Median Progression‑Free Survival (PFS) | 6.0 months |
| Median Overall Survival (OS) | 20.7 months |
FIRST‑08 is a multicenter, open‑label, single‑arm pivotal Phase II trial conducted in China, with results presented at the 2026 ASCO Annual Meeting. As of the 27 Dec 2025 data cutoff at a median follow‑up of 12 months, all 50 enrolled advanced cholangiocarcinoma patients had received at least one line of chemotherapy and one prior FGFR inhibitor, and 40 % had undergone three or more lines of systemic antitumor therapy – a heavily pretreated, historically refractory population. Efficacy endpoints were assessed by blinded independent central review (BICR).
Market Impact & Outlook
- The FGFR Resistance Dilemma: Cholangiocarcinoma is a highly aggressive biliary‑tract malignancy, with ≈25 % of patients harboring FGFR2 alterations. First‑generation FGFR inhibitors such as pemigatinib opened the door to targeted therapy, but virtually all patients eventually develop resistance – and until tinengotinib, no approved standard of care existed globally after FGFR‑inhibitor progression, leaving patients to fall back on chemotherapy or face a treatment void.
- From “No Drug Available” to Targeted Care: “FGFR2 fusion/rearrangement cholangiocarcinoma patients almost inevitably develop resistance after first‑generation FGFR inhibitor treatment, and previously lacked any approved option,” said Prof. Zhou Jun. “Tinengotinib’s entry into clinical practice gives these patients a targeted treatment choice for the first time.”
- Commercial Launch: Tinengotinib is TransThera’s first product transitioning from R&D to commercialization. COO Sun Delong stated the company will accelerate commercial deployment and access building so more drug‑resistant cholangiocarcinoma patients can benefit, while advancing development across additional indications and global markets.
- Patient Assistance: The Beijing Zhonghe Charity Foundation has launched the “Jiehu Ankang” assistance program; eligible advanced cholangiocarcinoma patients prescribed the drug by a clinician may apply for donated medication via the program platform (service hotline: 400‑013‑2468).
- Pipeline Expansion: Beyond the cholangiocarcinoma approval, tinengotinib is in Phase II trials across prostate cancer, breast cancer, and hepatocellular carcinoma, with a domestic Phase III confirmatory trial and an international multicenter Phase III registration trial progressing in parallel.
Forward‑Looking Statements
This brief contains forward‑looking statements regarding tinengotinib’s commercialization, indication expansion, ongoing Phase III trials, and global market development, as well as patient assistance program implementation. Actual results may differ materially due to risks including confirmatory trial outcomes, conversion of conditional approval to full approval, regulatory timelines, market adoption, and competitive dynamics. Conditional approval remains subject to completion of confirmatory requirements.-Fineline Info & Tech
