Sino Biopharmaceutical’s NDA for TQB3454, an oral IDH1 inhibitor, has been accepted by China’s CDE for advanced biliary tract cancer. Supported by a positive Phase III trial, the candidate could become the first IDH1‑targeted therapy approved in China
Written By: Nalam Karthik, PharmD
Reviewed By: Pharmacally Editorial Team
Sino Biopharmaceutical has submitted a New Drug Application to China’s National Medical Products Administration (NMPA) for TQB3454, an investigational oral IDH1 inhibitor, for the treatment of advanced biliary tract cancer (BTC) harboring IDH1 mutations. The application has been accepted by the Center for Drug Evaluation (CDE), marking a significant regulatory milestone for the company’s precision oncology program.
The candidate was independently developed by Chia Tai Tianqing Pharmaceutical Group Co., Ltd. (CTTQ), a subsidiary of Sino Biopharmaceutical. The CDE granted Breakthrough Therapy Designation to TQB3454 in April 2023 based on its clinical potential and later included the application in the Priority Review and Approval pathway in May 2026.
TQB3454 Targets a Key Oncogenic Driver
TQB3454 selectively inhibits the mutant isocitrate dehydrogenase 1 (IDH1) enzyme, a genetic alteration found in a subset of biliary tract cancers, particularly intrahepatic cholangiocarcinoma. Mutant IDH1 produces the oncometabolite 2-hydroxyglutarate (2-HG), which disrupts normal cellular differentiation through widespread DNA and histone hypermethylation.
By blocking mutant IDH1 activity, TQB3454 lowers 2-HG levels, reverses these epigenetic abnormalities, restores normal chromatin function, and promotes the differentiation of malignant cells, ultimately suppressing tumor growth.
Phase III Trial Showed Survival Benefit
The NDA is supported by results from the randomized, double-blind, placebo-controlled, multicenter Phase III TQB3454-III-01 trial (NCT05987358), which evaluated the therapy in patients with advanced IDH1-mutant biliary tract cancer whose disease had progressed after gemcitabine- and fluoropyrimidine-based chemotherapy.
According to the company, TQB3454 significantly reduced the risk of disease progression or death compared with placebo. The study also demonstrated statistically meaningful improvements in both progression-free survival (PFS) and overall survival (OS). Detailed efficacy data, including hazard ratios and confidence intervals, were not disclosed in the announcement.
The treatment’s safety profile was consistent with previous clinical experience, and investigators reported no new safety signals, supporting a favorable benefit-risk profile.
The company noted that this represents the first positive Phase III study of a domestically developed IDH1 inhibitor in biliary tract cancer and only the second Phase III trial globally to demonstrate positive outcomes for this therapeutic approach.
High Unmet Need in Biliary Tract Cancer
Biliary tract cancer includes intrahepatic, perihilar, and distal cholangiocarcinoma, as well as gallbladder cancer, and accounts for approximately 3% of gastrointestinal malignancies. Most cases are adenocarcinomas with an aggressive clinical course and a 5-year survival rate below 5%.
The incidence of BTC continues to rise worldwide, with the highest rates reported across Asia. IDH1 mutations occur in approximately 4.9% to 20% of patients with intrahepatic cholangiocarcinoma, creating a defined subgroup that may benefit from targeted therapy.
Regulatory Path
If approved, TQB3454 could become the first IDH1-targeted therapy approved in China for biliary tract cancer, addressing an important treatment gap for patients with previously treated IDH1-mutant BTC. The Priority Review designation may also accelerate the regulatory assessment, potentially bringing a new precision medicine option to eligible patients sooner.
Reference
ACCEPTANCE OF NEW DRUG APPLICATION FOR TQB3454 TABLET “IDH1 INHIBITOR
About the Writer
Nalam Karthik (LinkedIn) is a healthcare writer and PharmD graduate with interests in pharmacovigilance, drug safety, clinical data analysis, and quality assurance. He is passionate about translating clinical and pharmaceutical knowledge into accessible healthcare content while staying engaged with advancements in drug development and patient safety initiatives.