Orphan Drug Status for Loxo Oncology Tumor Drug

FDA has granted Loxo Oncology an orphan drug designation for larotrectinib for treating solid tumors with NTRK-fusion proteins, which are genetic abnormalities that occur rarely in various adult and pediatric solid tumors. Larotrectinib (LOXO-101) is a potent, oral and selective investigational new drug in clinical development for treating patients with cancers that harbor abnormalities involving the tropomyosin receptor kinases (TRKs). “Growing research suggests that the NTRK genes, which encode for TRKs, can become abnormally fused to other genes, resulting in growth signals that can lead to cancer in many sites of the body,” the company says. The therapy is also being evaluated in the NAVIGATE global Phase 2 multi-center trial in patients with solid tumors that harbor TRK gene fusions, and the SCOUT Phase 1/2 trial in pediatric patients, including patients with advanced cancer, TRK gene fusions and infantile fibrosarcoma.