At the 2025 Annual Congress of the International Society of Paediatric Oncology (SIOP), clinical data from Phase I/II trials of Zolbetinib—a domestically developed next-generation pan-TRK inhibitor—were unveiled. The trials focused on its efficacy in treating advanced solid tumors in children and adolescents. The findings revealed that Zolbetinib exhibited a manageable safety profile and potent antitumor activity in pediatric patients with solid tumors harboring NTRK gene fusions. This positions it as a promising therapeutic option for malignancies driven by NTRK gene alterations, with the added advantage of overcoming resistance to first-generation TRK inhibitors.

NTRK gene fusion-positive tumors are more prevalent in children than in adults, and conventional treatments have demonstrated limited effectiveness against them. As a second-generation TRK inhibitor, Zolbetinib adopts a "one-drug-fits-multiple-cancers" strategy, achieving a high objective response rate while maintaining a favorable safety profile among the pediatric cohort enrolled in the trials.

Identifying NTRK gene fusion positivity in children necessitates molecular pathology testing, such as next-generation sequencing (NGS). While such testing is readily accessible at major cancer centers across the country, its availability remains constrained in grassroots medical institutions.

Looking ahead, the potential for combining Zolbetinib with other therapeutic approaches warrants further exploration. Recently, the drug has been integrated into the "Starlight Program," fueling expectations that this innovative, domestically produced medication will soon bring relief to a broader spectrum of pediatric cancer patients.