Abstract
Larotrectinib is a highly selective inhibitor of the TRK receptor tyrosine kinase domain that blocks the downstream signalling pathway of the TRK receptor. It is indicated for the treatment of adult and paediatric patients with advanced solid tumours harbouring NTRK gene fusions.
The efficacy and safety of larotrectinib has been demonstrated in three phase I–II clinical trials enrolling patients with 17 primary tumour types. The treatment with larotrectinib led to a high objective response rate (responses were rapid and durable) as well as the prolongation of progression-free survival and overall survival.
In most of the patients, treatment with larotrectinib was associated with improvement, or at least maintenance of quality of life. Larotrectinib showed a good safety profile.
Thus, it is a very promising agent, however, the identification of patients with NTRK gene fusions remains challenging.