Efficacy (paediatrics)
Overall, in the paediatric sub-population (n=70) of the pooled analysis the ORR was 87% [1]. Data from the phase 1/2 SCOUT trial in paediatric patients and adolescents/young adults with advanced solid or primary CNS tumours were analysed separately and included patients with and without identified NTRK gene fusions. The ORR was 93% (14/15) in evaluable patients with cancers with TRK fusion cancer, compared with 0% (0/7) without TRK fusion cancer. Larotrectinib was detectable in the cerebrospinal fluid (CSF) of two patients that underwent standard-of-care sampling with CSF to plasma concentrations of 28% via Ommaya and 123% via lumbar puncture.
References
- VITRAKVI® (larotrectinib), EMA Summary of medicinal Product Characteristics. 2022.