FDA accepted and granted Priority Review to an NDA for larotrectinib (LOXO-101) from Loxo Oncology Inc. (NASDAQ:LOXO) to treat locally advanced or metastatic solid tumors with neurotrophic tyrosine kinase receptor (Trk) fusion proteins, regardless of tissue of origin. Its PDUFA date is Nov. 26.
Loxo's development program for larotrectinib was designed specifically to enroll a genetically defined patient population. The therapy has breakthrough therapy, rare pediatric disease and Orphan Drug designations from FDA (see BioCentury, June 13, 2016).
Larotrectinib is a small molecule inhibitor of neurotrophic tyrosine kinase receptor 1 (TrkA; NTRK1), TrkB (NTRK2) and TrkC (NTRK3). Loxo said the therapy led to a centrally assessed overall response rate (ORR) of 75%, and an investigator-assessed confirmed ORR of 80%, among 55 evaluable patients with NTRK gene fusions across various solid tumor types in three Phase I and Phase II trials.
Bayer AG (Xetra:BAYN), which shares larotrectinib's rights, plans to submit an MAA to EMA for the therapy this year (see BioCentury Extra, Nov. 14, 2017).
One other oncology drug has received FDA approval in a biomarker-specific indication, regardless of tissue type. Keytruda pembrolizumab from Merck & Co. Inc. (NYSE:MRK), a mAb against PD-1, has accelerated approval to treat advanced microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) solid tumors.
Loxo gained $2.68 to $168.39 on Tuesday.