FDA

Vitrakvi granted full approval for NTRK gene fusion-positive solid tumors

April 14, 2025

FDA granted full approval to Vitrakvi (larotrectinib) for the treatment of adult and pediatric patients with solid tumors that have a neurotrophic receptor tyrosine kinase (NTRK) gene fusion without a known acquired resistance mutation, are metastatic or where surgical resection is likely to result in severe morbidity, and have no satisfactory alternative treatments or that have progressed following treatment.

Efficacy

Full approval was based on data from three multicenter, open-label, single-arm clinical trials: LOXO-TRK-14001 (NCT02122913), SCOUT (NCT02637687), and NAVIGATE (NCT02576431). The analysis included 339 pediatric and adult patients with unresectable or metastatic solid tumors with an NTRK gene fusion. The major efficacy outcome measures were overall response rate (ORR) and duration of response (DOR). Safety was assessed in 444 patients across the three trials.

Pooled efficacy results demonstrated an ORR of 60% (95% confidence interval [CI], 55%-65%) with a complete response (CR) rate of 24% and a partial response (PR) rate of 36%. Median DOR was 43.3 months (95% CI, 32.5-not evaluable).

Safety

The most common (≥20%) adverse reactions with Vitrakvi were increased AST, increased ALT, anemia, hypoalbuminemia, musculoskeletal pain, increased alkaline phosphatase, leukopenia, lymphopenia, neutropenia, hypocalcemia, fatigue, vomiting, cough, constipation, pyrexia, diarrhea, nausea, abdominal pain, dizziness, and rash.

Serious adverse events reported include CNS problems, bone fractures, and hepatoxicity.

Sources:

US FDA grants full approval of Vitrakvi® (larotrectinib) for adult and pediatric patients with NTRK gene fusion-positive solid tumors. [News release]. 2025. https://www.bayer.com/en/us/news-stories/approval-of-vitrakvi

Vitrakvi (larotrectinib) [package insert]. U.S. Food and Drug Administration. https://labeling.bayerhealthcare.com/html/products/pi/vitrakvi_PI.pdf Revised April 2025. Accessed April 11, 2025.