Zai Lab's AUGTYRO™ Approved for ROS1-Positive NSCLC by China's NMPA

Zai Lab Limited has announced that China's National Medical Products Administration (NMPA) has approved the New Drug Application for AUGTYRO™ (repotrectinib). This approval is intended for adult patients with locally advanced or metastatic ROS1-positive non-small-cell lung cancer (NSCLC). The decision is based on the results from the TRIDENT-1 trial, which demonstrated significant efficacy of repotrectinib in both TKI-naïve and TKI-pretreated patients with ROS1-positive NSCLC.

Dr. Rafael G. Amado, President and Head of Global Oncology Research and Development at Zai Lab, emphasized the importance of this approval, noting the substantial unmet medical need due to the resistance developed against existing therapies. Dr. Shun Lu, Chief of Lung Cancer Center at Shanghai Chest Hospital, also underscored the promising results of the TRIDENT-1 study, which showed high response rates and durability in ROS1-positive NSCLC patients.

The TRIDENT-1 trial, a global, open-label, single-arm, Phase 1/2 study, was pivotal in the approval process. It assessed the safety and efficacy of repotrectinib in patients with advanced solid tumors harboring ROS1 fusions. The trial included both TKI-naïve and TKI-pretreated patients and allowed those with asymptomatic CNS metastases. Key findings from the trial showed a 79% response rate in TKI-naïve patients and a 38% response rate in TKI-pretreated patients. The study also noted significant intracranial responses.

Zai Lab played a crucial role in the TRIDENT-1 study, enrolling the first patient in Greater China in May 2021. The results, aligned with global data, were published in the New England Journal of Medicine in January 2024. AUGTYRO demonstrated robust response rates and durable clinical activity, with a manageable safety profile.

AUGTYRO is a next-generation tyrosine kinase inhibitor designed to target ROS1 and NTRK oncogenic drivers. It aims to overcome resistance mutations that often limit the efficacy of current therapies, especially in the brain. The drug has received various designations from the U.S. FDA, including Breakthrough Therapy and Fast-Track, for different subsets of patients with ROS1-positive and NTRK-positive cancers.

In June 2023, China's NMPA accepted the NDA for AUGTYRO, granting priority review in May 2023. The approval marks a significant milestone for Zai Lab, which has an exclusive license agreement with Turning Point Therapeutics, a Bristol Myers Squibb company, to develop and commercialize AUGTYRO in Greater China.

Non-small-cell lung cancer is a major health concern in China, with about 871,000 new cases and 767,000 deaths reported in 2022. NSCLC comprises roughly 85% of all lung cancer cases, with ROS1 rearrangements occurring in 2-3% of advanced NSCLC patients.

Zai Lab, based in China and the United States, focuses on oncology, autoimmune disorders, infectious diseases, and neuroscience. The company aims to address significant unmet medical needs through innovative research and development.