China Approves Yochanra (Tinengotinib) for FGFR Inhibitor-Resistant Cholangiocarcinoma

China’s National Medical Products Administration (NMPA) has approved Yochanra® (tinengotinib) for adults with advanced, metastatic, or unresectable cholangiocarcinoma harboring an FGFR2 fusion or rearrangement who have previously received systemic therapy and an FGFR inhibitor. Granted on August 6, 2026, the approval makes Yochanra the first drug independently developed in China specifically addressing acquired resistance to FGFR inhibitor therapy in this molecularly defined patient population.

Phase II Data Support NMPA Approval

The approval was based on a multicenter, open-label, single-arm pivotal Phase II study (NCT06057571) conducted in China. The trial enrolled 50 patients with advanced cholangiocarcinoma who had previously received at least one line of chemotherapy and treatment with an FGFR inhibitor. The results were presented at the 2026 American Society of Clinical Oncology (ASCO) Annual Meeting.

At a median follow-up of 12 months, blinded independent central review showed an objective response rate (ORR) of 28.0%, with 14 patients achieving confirmed partial responses. The median duration of response (DoR) was 8.5 months (95% CI, 5.6-12.5), while the disease control rate (DCR) reached 82.0%.

Median progression-free survival (PFS) was 6.0 months (95% CI, 4.4-8.3), and median overall survival (OS) was 20.7 months (95% CI, 11.8-not estimable). The company reported that tinengotinib demonstrated durable antitumor activity with a manageable safety and tolerability profile.

Addressing Acquired FGFR Inhibitor Resistance

FGFR2 fusions and rearrangements occur in a subset of patients with cholangiocarcinoma and have emerged as actionable molecular targets. However, patients treated with FGFR inhibitors can develop acquired resistance, often through secondary alterations in the FGFR2 kinase domain. These resistance mechanisms can limit the duration of benefit from currently available targeted therapies and leave patients with limited established treatment options after progression.

Tinengotinib is an oral multi-target small-molecule kinase inhibitor that targets FGFR and VEGFR as well as JAK and Aurora kinases. Its development in cholangiocarcinoma focuses on its potential to retain activity against multiple FGFR resistance mutations while maintaining inhibition of FGFR signaling.

This broader activity distinguishes its development strategy from selective FGFR inhibition and provides the biological rationale for evaluating tinengotinib after progression on prior FGFR inhibitor therapy.

Global Regulatory and Phase III Development

Tinengotinib has received Fast Track and Orphan Drug Designations from the U.S. Food and Drug Administration (FDA) for cholangiocarcinoma. The European Medicines Agency (EMA) also granted Orphan Drug Designation for biliary tract cancer. In China, the program previously received Breakthrough Therapy Designation and was included in the priority review pathway.

Global development is continuing through the randomized Phase III FIRST-308 study (NCT05948475), which is evaluating tinengotinib against physician’s choice of chemotherapy in patients with FGFR-altered cholangiocarcinoma following prior FGFR inhibitor therapy. The comparator regimen includes FOLFOX or FOLFIRI.

The company has also completed enrollment in the global multicenter registrational Phase III program for cholangiocarcinoma, while a confirmatory Phase III study in China is progressing in parallel.

Expansion Beyond Cholangiocarcinoma

The NMPA decision represents the first approved indication for Yochanra under a broader multi-indication development strategy. Clinical development is also evaluating tinengotinib in other solid tumors, including prostate cancer, breast cancer, and liver cancer.

The company is investigating whether tinengotinib’s activity across multiple kinase pathways can translate into clinical benefit in tumors with distinct molecular drivers and treatment-resistance mechanisms.

For patients with FGFR2 fusion- or rearrangement-positive cholangiocarcinoma whose disease has progressed after FGFR inhibitor therapy, the approval provides a new treatment option in a setting where established post-FGFR inhibitor standards remain limited. The ongoing Phase III studies will determine whether the activity observed in Phase II can support a broader role for tinengotinib in the global treatment of FGFR-altered cholangiocarcinoma.

 Reference

Yochanra®, the World’s First Innovative Drug to Overcome FGFR Inhibitor Resistance, Makes Its Global Debut in China – TransThera Sciences (Nanjing), Inc. — Transformative Therapy! TransThera