Zai Lab’s Repotrectinib Receives NMPA Acceptance for Treating NTRK-Positive Solid Tumors in China

Zai Lab Limited, a biopharmaceutical company listed on both NASDAQ and the Hong Kong Stock Exchange (HKEX), has announced that China’s National Medical Products Administration (NMPA) has accepted the supplemental New Drug Application (sNDA) for repotrectinib. This drug is intended to treat adult patients with solid tumors that have a neurotrophic tyrosine receptor kinase (NTRK) gene fusion. The sNDA targets patients whose cancer is locally advanced or metastatic, or where surgery could result in severe morbidity, and who have either progressed after prior therapies or have no other suitable treatment options. The acceptance of this application brings repotrectinib one step closer to becoming the first approved treatment for NTRK-positive cancers in China, covering both TKI-naïve and TKI-pretreated patients across a range of solid tumors. NTRK fusion-positive tumors are a type of cancer where an NTRK gene has merged with another gene, leading to the production of an abnormal NTRK fusion protein that drives tumor growth. These tumors are rare but can occur in various types of cancer, including lung, breast, and colon cancers. They are particularly challenging because they often develop resistance to existing TRK tyrosine kinase inhibitors (TKIs), leading to tumor progression and poor patient outcomes. Repotrectinib is designed to overcome this resistance and provide a more durable response, even in tumors that have spread to the brain. The journey of repotrectinib to this point has been marked by significant milestones. In February 2025, the NMPA granted repotrectinib priority review for the treatment of advanced solid tumors with NTRK gene fusions. This fast-track designation underscores the potential of repotrectinib to address a critical unmet need in oncology. Prior to this, in June 2024, the U.S. Food and Drug Administration (FDA) approved repotrectinib, marketed as AUGTYRO, for the treatment of adult and pediatric patients aged 12 years and older with NTRK-positive solid tumors. The FDA had previously approved AUGTYRO for adult patients with locally advanced or metastatic ROS1-positive non-small-cell lung cancer (NSCLC) in November 2023, highlighting its broad therapeutic potential. Repotrectinib is a next-generation tyrosine kinase inhibitor (TKI) that targets ROS1 and NTRK oncogenic drivers. Unlike first-generation TKIs, repotrectinib is uniquely designed to maintain its effectiveness even when resistance mutations develop. This is crucial because current treatments often lead to a shortened duration of response and tumor progression due to the emergence of these mutations. Repotrectinib's design allows it to penetrate the brain more effectively, making it a promising treatment for brain metastases, a common complication in many cancers. Dr. Rafael Amado, President and Head of Global Research and Development at Zai Lab, emphasized the significance of this development: "NTRK fusion-positive tumors represent a significant therapeutic challenge, especially with acquired resistance to existing TRK TKIs. Given that there are currently no approved treatments for NTRK-positive cancers in both TKI-naïve and TKI-pretreated patients in China, repotrectinib has the potential to make a substantial impact." Zai Lab has an exclusive license agreement with Turning Point Therapeutics (now part of Bristol Myers Squibb) to develop and commercialize repotrectinib in Greater China, which includes mainland China, Hong Kong, Taiwan, and Macau. This partnership is crucial for Zai Lab’s strategy to bring innovative treatments to Chinese patients and to expand its portfolio in oncology. NTRK-positive advanced malignancies are life-threatening and have poor prognoses, presenting a significant unmet medical need. While existing targeted therapies have shown some clinical benefits, their effectiveness is limited by the emergence of resistance mutations. In China, there has been a particular gap in treatment options for these patients, making the acceptance of the sNDA for repotrectinib a significant step forward. Zai Lab is an innovative, research-based, commercial-stage biopharmaceutical company with a focus on developing and commercializing products to address significant unmet medical needs in oncology, immunology, neuroscience, and infectious diseases. The company's broader portfolio and strategic partnerships, such as the one with Turning Point Therapeutics, position it well to continue making a positive impact on patient health. However, the company also acknowledges the risks and uncertainties inherent in drug development, including regulatory approvals and business operations in China. Industry insiders view the acceptance of the sNDA by the NMPA as a pivotal moment for Zai Lab and the oncology community in China. It highlights the company's commitment to bringing cutting-edge treatments to a market with limited options. Zai Lab’s extensive experience in drug development and its strategic focus on addressing unmet needs make it a strong player in the biopharmaceutical sector. The potential approval of repotrectinib could significantly improve treatment outcomes for patients with NTRK-positive solid tumors, both in China and globally.