EU Approves TransThera's Phase 3 Cholangiocarcinoma Trial and Tinengotinib's Orphan Drug Status

TransThera, a biopharmaceutical firm in the clinical stage, has announced that the Phase 3 global clinical trial for tinengotinib has received approval from the European Union. This follows previous authorizations in the United States, South Korea, and Taiwan. The trial, known as FIRST-308, is a randomized and controlled study that compares tinengotinib with the physician's choice of treatment for patients with cholangiocarcinoma (CCA) that has been altered by FGFR, and has been refractory or relapsed after chemotherapy and FGFR inhibitor treatment.

Tinengotinib, classified as a next-generation FGFR inhibitor, has demonstrated significant clinical benefits, particularly for patients with metastatic CCA that has been heavily pre-treated with chemotherapy and is resistant to FGFR inhibitors. The drug has shown its potential in phase I/II trials, with results being presented at the 2023 ESMO and 2024 ASCO GI conferences. The FIRST-308 trial is currently enrolling participants to further validate the drug's efficacy and safety in treating CCA. The first patient was administered the drug in the United States in December 2023.

Jean Fan, M.D., Chief Medical Officer at TransThera Sciences, expressed enthusiasm about the regulatory advancements for tinengotinib, highlighting the global recognition of its potential to benefit CCA patients. He emphasized the company's dedication to delivering innovative treatments to patients worldwide and the acceleration of the drug's global clinical development and commercialization.

In the European Union, "orphan medicines" are pharmaceutical products intended for rare conditions affecting fewer than 5 in 10,000 people. The EMA provides incentives for the development of such medicines, including support during clinical development, reduced fees, and a 10-year market exclusivity period post-approval.

Tinengotinib is a novel, phase III spectrum-selective kinase inhibitor that targets various pathways involved in tumor growth, including cytokine signaling, angiogenesis, and immune-oncology pathways. It has shown promise in treating various solid tumors, and has received Orphan Drug Designation and Fast Track Designation from the FDA for CCA treatment. Additionally, it was granted Breakthrough Therapy Designation by China's NMPA in July 2023 and ODD for biliary tract cancer by EMA in March 2024.

TransThera Sciences is a clinical-stage company that focuses on developing innovative therapeutics for diseases with significant unmet medical needs. Their portfolio includes treatments for oncology, inflammatory, and cardiovascular diseases.