Zai Lab's Repotrectinib sNDA for NTRK-Positive Tumors Accepted

Zai Lab Limited has announced that China's National Medical Products Administration (NMPA) has accepted a new drug application for repotrectinib. This application targets adult patients with solid tumors that have a neurotrophic tyrosine receptor kinase (NTRK) gene fusion. This treatment is aimed at those with locally advanced or metastatic conditions, or where surgery could lead to severe complications, especially in patients who have not responded to previous therapies or lack suitable treatment alternatives.

Dr. Rafael Amado, President and Head of Global Research and Development at Zai Lab, highlighted the challenges posed by NTRK fusion-positive tumors, particularly due to resistance developed against current TRK tyrosine kinase inhibitors (TKIs). In China, there is a lack of approved treatments for both TKI-naïve and TKI-pretreated patients suffering from NTRK-positive cancers. Repotrectinib is seen as a promising next-generation TKI that could potentially treat a wide range of NTRK fusion-positive tumors in these scenarios.

Earlier, in February 2025, the NMPA had given priority review status to repotrectinib for addressing advanced solid tumors with an NTRK gene fusion. Repotrectinib acts as a next-generation tyrosine kinase inhibitor, targeting ROS1 and NTRK oncogenic drivers. Patients with ROS1 and NTRK gene fusion solid tumors often develop mutations that resist current therapies, leading to shorter response durations and tumor progression. Repotrectinib is designed to enhance durability of response, even in cases involving the brain, and to manage acquired resistance.

In June 2024, the U.S. Food and Drug Administration (FDA) approved repotrectinib, branded as AUGTYRO, for treating adult and pediatric patients aged 12 and older with solid tumors harboring an NTRK gene fusion. This approval covers cases that are either locally advanced or metastatic, where surgery might cause severe morbidity, and where patients show progression after treatment or have no effective treatment alternatives.

Additionally, in May 2024, the NMPA approved AUGTYRO for adult patients with locally advanced or metastatic ROS1-positive non-small-cell lung cancer (NSCLC). This followed an earlier approval by the FDA for the same indication in November 2023. Zai Lab holds an exclusive license agreement with Turning Point Therapeutics, Inc., a Bristol Myers Squibb company, to develop and market AUGTYRO in Greater China, encompassing mainland China, Hong Kong, Taiwan, and Macau.

NTRK-positive solid tumors, which pose a significant threat due to poor prognoses, represent an area of considerable unmet medical need in both adult and pediatric patients. While existing targeted therapies have shown some clinical benefits, their effectiveness is limited by the emergence of resistance mutations. In China, there has been no approved treatment for NTRK-positive cancers studied in both TKI-naïve and TKI-pretreated patients across various solid tumors.

Zai Lab Limited is a biopharmaceutical company operating out of China and the United States, focusing on developing and commercializing innovative products in oncology, immunology, neuroscience, and infectious diseases. The company aims to address medical conditions with significant unmet needs and positively impact human health through leveraging its competencies and resources.