Aiming at the target works. This is demonstrated by six analyzes of larotrectinib, a drug effective in patients, adults and children, with tumors that present a specific alteration, an NTRK fusion gene (also called tumors with TRK fusion). The analyzes were presented at the ASCO conference in Chicago and include efficacy and safety data from 244 adults and children in 25 different tumor types and a separate subgroup analysis of 94 children with TRK fusion solid tumors with an age median of 2.2 years. Both patient groups at increased follow-up continue to demonstrate that larotrectinib offers rapid and lasting responses, a sustained survival benefit, and a favorable safety profile in patients with TRK fusion solid tumors.
The potent efficacy and safety benefits of larotrectinib treatment are also confirmed in an extended cohort of patients with TRK fusion lung cancer, including those with CNS metastases. Larotrectinib achieved a confirmed objective response rate of 83%, rapid and durable responses, prolonged survival with overall survival rates at 24 and 36 months both of 72% (at a median follow-up of 12.9 months), and a favorable long-term safety profile.
A specific subgroup analysis of adults and children with primary central nervous system tumors with TRK fusion shows that larotrectinib achieves a high rate of disease control, rapid and lasting responses, and a manageable safety profile in these patients. Median overall survival was not achieved at the median follow-up of 26.7 months. The overall survival rate at 24 months was 65%.
In a separate analysis of children with non-CNS solid tumors with TRK fusion treated with larotrectinib, the investigator-assessed objective response rate was 84% for the 93 evaluable patients, with 37% complete responses. .
In a separate analysis of a subgroup of patients with primary central nervous system tumors, larotrectinib achieved a high rate of disease control, rapid and lasting responses, and a favorable safety profile.
Finally, the update of the health-related quality of life data then demonstrates that patients with TRK fusion solid tumors treated with larotrectinib continue to show rapid and clinically significant improvements in quality of life. The results of the indirect comparison suggest that adult patients with TRK fusion solid tumors treated with larotrectinib exhibit greater overall survival than patients treated with standard of care. These findings underscore the importance of identifying adult and pediatric patients with NTRK fusion solid tumors, through the broad offering of patient access to comprehensive genomic cancer testing.