Larotrectinib was initially awarded
orphan drug status in 2015, for soft tissue sarcoma, and
breakthrough therapy designation in 2016 for the treatment of metastatic solid tumors with NTRK fusion.[10] Some clinical trial results were announced in 2017.[11] On 26 November 2018, Larotrectinib was approved by the FDA.[12]
Larotrectinib was the first drug to be specifically developed and approved to treat any cancer containing certain mutations, as opposed to cancers of specific tissues (i.e., the approval is "
tissue agnostic"). Several earlier drugs, including
pembrolizumab, were eventually approved by the FDA for treatment of specific mutations independent of the type of cancer, but those drugs had been initially developed for specific cancer types.[13] The U.S.
Food and Drug Administration (FDA) considers it to be a
first-in-class medication.[14]
Phase II clinical trials evaluating the drug for efficacy and safety in treating several types of solid tumors are ongoing.[15]
Larotrectinib was approved for medical use in the European Union in September 2019.[16][17] It was approved for medical use in Australia in August 2020.[4]
Recent publications have also highlighted advancements in optimizing the manufacturing process of
Larotrectinib using
continuous flow chemistry, improving its scalability and purity.[18]
^"Larotrectinib"(PDF). Statement on a Nonproprietary Name Adopted by the USAN Council. American Medical Association (AMA). 26 October 2016.
^Berger S, Martens UM, Bochum S (2018). "Larotrectinib (LOXO-101)". Small Molecules in Oncology. Recent Results in Cancer Research Fortschritte der Krebsforschung. Progres dans les Recherches Sur le Cancer. Vol. 211. pp. 141–151.
doi:
10.1007/978-3-319-91442-8_10.
ISBN978-3-319-91441-1.
PMID30069765.
^Clinical trial number NCT02576431 for "A Phase 2 Basket Study of the Oral TRK Inhibitor Larotrectinib in Subjects With NTRK Fusion-positive Tumors" at
ClinicalTrials.gov