The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has recommended Bayer’s larotrectinib for marketing authorization in the European Union (EU).
The recommended indication is the treatment of adult and peadiatric patients with solid tumours that display a Neurotrophic Tyrosine Receptor Kinase (NTRK) gene fusion, who have a disease that is locally advanced, metastatic or where surgical resection is likely to result in severe morbidity, and who have no satisfactory treatment options.
The final decision of the European Commission on the marketing authorization is expected in the coming months.
Larotrectinib, a first-in-class oral TRK inhibitor specifically developed to treat tumours that have an NTRK gene fusion, will be the first treatment to receive a tumour-agnostic indication in the EU. Larotrectinib is already approved in the US as well as in Brazil and Canada.
The CHMP recommendation is based on pooled clinical trial data of 102 patients (93 patients from the primary analysis population and an additional 9 patients with primary central nervous [CNS] tumours) across the phase I trial of adult patients, the phase II NAVIGATE trial in adult and adolescent patients and the phase I/II pediatric SCOUT trial, showing a high response rate with durable and rapid responses for larotrectinib.
Results in the primary analysis population demonstrate an overall response rate (ORR) of 72% (95% CI: 62, 81), including a 16% complete response (CR) rate and a 55% partial response (PR) rate. In an additional analysis including primary CNS patients, the ORR was 67% (95% CI: 57, 76), including a 15% CR, and a 51% PR.
TRK fusion cancer is rare overall, affecting no more than a few thousand patients across Europe annually. It affects both children and adults and occurs in varying frequencies across various tumour types.
In clinical trials, larotrectinib was investigated across 29 different histologies of solid tumours including lung, thyroid, melanoma, gastrointestinal stromal tumours, colon, soft tissue sarcomas, salivary gland and infantile fibrosarcoma.