The European Commission has granted conditional marketing authorisation in the European Union (EU) for larotrectinib, which is indicated for the treatment of adult and paediatric patients with solid tumours that display neurotrophic tyrosine receptor kinase (NTRK) gene fusion, who have locally advanced or metastatic disease, or where surgical resection is likely to result in severe morbidity, and who have no satisfactory treatment options.
The first-in-class oral TRK inhibitor is the first treatment in the EU to receive a tumour-agnostic indication.
The licence is based on pooled data for 102 patients from a phase 1 trial of adults, the phase 2 NAVIGATE trial in adults and adolescents and the phase 1/2 paediatric SCOUT trial.
The primary analysis population demonstrated an overall response rate (ORR) of 72 per cent (95% CI 62%-81%). In an additional analysis including primary central nervous system (CNS) patients, the ORR was 67 per cent (95% CI 57%-76%). In the pooled primary analysis set, neither the median duration of response nor median progression-free survival had been reached at the time of analysis.
Larotrectinib showed a favourable safety profile, with the majority of adverse events (AEs) being grade 1 or 2. Three per cent of patients permanently ceased therapy due to treatment-emergent AEs.