China's National Medical Products Administration (NMPA) has granted conditional marketing approval to Yochanra (tinengotinib) tablets for adult patients with unresectable advanced or metastatic cholangiocarcinoma (CCA) harboring fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement who have received prior systemic therapy and FGFR inhibitor treatment. The approval, announced by China-based TransThera Sciences (Nanjing), Inc. (HKEX: 2617), marks the first approved therapy in China specifically for patients who have progressed on an FGFR inhibitor — a population that previously had no approved treatment options.
The conditional approval is supported by a multicenter, open-label, single-arm pivotal Phase II trial conducted in China. Among 50 heavily pretreated patients with advanced CCA — all of whom had received at least one prior line of chemotherapy and one prior FGFR inhibitor — blinded independent central review (BICR) assessment at a median follow-up of 12 months reported an objective response rate (ORR) of 28.0%, with a median duration of response (DoR) of 8.5 months (95% CI: 5.6–12.5). The disease control rate (DCR) was 82.0%, median progression-free survival (mPFS) was 6.0 months (95% CI: 4.4–8.3), and median overall survival (mOS) was 20.7 months (95% CI: 11.8–not reached). A Phase III confirmatory study versus chemotherapy is ongoing in China.
Tinengotinib is a multi-kinase inhibitor targeting FGFR1/2/3, JAK1/2, VEGFR2, and Aurora A/B kinases. Unlike selective FGFR inhibitors, its broader kinase profile is intended to retain activity against acquired resistance mutations such as FGFR2 V564F.
The approved indication occupies a distinct clinical niche from existing FGFR inhibitors. Pemigatinib, approved by the NMPA in 2022, targets FGFR2-altered CCA in patients who have received prior systemic therapy but not necessarily a prior FGFR inhibitor, placing it earlier in the treatment sequence. The evolving CCA targeted therapy landscape now includes multiple molecularly defined subgroups, with HUTCHMED's fanregratinib also pursuing NMPA approval in FGFR2 fusion-positive CCA. Globally, Relay Therapeutics' lirafugratinib — a covalent, FGFR2-selective inhibitor also designed for post-FGFR inhibitor progression — has an FDA new drug application under Priority Review with a PDUFA target date of September 27, 2026.