Drugs / Larotrectinib
last change Dec 2025 re-read 2 minutes ago

Larotrectinib

Small molecule targets NTRK1, NTRK2, NTRK3 via inhibition

Developed for
NTRK fusion positive cancer · ependymoma · hepatoblastoma · Langerhans cell histiocytosis · lung carcinoma · malignant germ cell tumor · malignant glioma · medulloblastoma
+28 more · neuroblastoma · non-Hodgkin lymphoma · plasma cell myeloma · rhabdoid tumor · rhabdomyosarcoma · thyroid gland carcinoma · acute leukemia · adult lymphoma · central nervous system neoplasm · colorectal cancer · conventional osteosarcoma · differentiated thyroid carcinoma · diffuse intrinsic pontine glioma · Ewing sarcoma · Ewing sarcoma/peripheral primitive neuroectodermal tumor · histiocytic sarcoma · juvenile xanthogranuloma · kidney Wilms tumor · metastatic malignant neoplasm · osteosarcoma · pediatric fibrosarcoma · peripheral primitive neuroectodermal tumor · primary organ-specific lymphoma · refractory malignant neoplasm · salivary gland carcinoma · soft tissue sarcoma · thyroid tumor · Wilms tumor
Investigated by
National Cancer Institute (NCI) · Bayer · Sun Yat-sen University · Changhai Hospital · Children's Hospital of Philadelphia · Children's Oncology Group
+34 more · Dalian Friendship Hospital Affiliated to Dalian Medical University · Fred Hutchinson Cancer Center · Fudan University · Fujian Cancer Hospital · Fujian Medical University · Henan Cancer Hospital · Hunan Provincial People's Hospital · Jiangsu Cancer Institute & Hospital · Liaoning Cancer Hospital & Institute · M.D. Anderson Cancer Center · Nationwide Children's Hospital · Northwell Health · Oregon Health and Science University · Oslo University Hospital · Peking Union Medical College · Peking Union Medical College Hospital · Peking University Hospital of Stomatology · Presage Biosciences · Ruijin Hospital · Shanghai 10th People's Hospital · Shanghai 6th People's Hospital · Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine · Shanghai Ninth People's Hospital Affiliated to Shanghai Jiao Tong University · Shanxi Province Cancer Hospital · Sir Run Run Shaw Hospital · Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University · The Affiliated Hospital of Inner Mongolia Medical University · The First Affiliated Hospital of Anhui University of Chinese Medicine · The Third Affiliated Hospital of Soochow University · Tianjin Medical University Cancer Institute and Hospital · Union Hospital, Tongji Medical College, Huazhong University of Science and Technology · University of Washington · West China Hospital · Zhejiang Cancer Hospital

Regulatory milestones approvals, filings & regulatory actions · 4 recorded

MilestoneJurisdictionBrandIndicationDateSentence it was read from
Label expansions (2) US (FDA) VITRAKVI 2021-03-25 – 2025-04-09 fda.gov
Approved · conditional EU (EMA) Vitrakvi 2019-09-19 europa.eu
Approved US (FDA) VITRAKVI 2018-11-26 fda.gov

Trials 19 · a red edge is where a trial was stopped

PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 01 trial
Phase 0 NCT04655404 Apr 2021 → Dec 2026 expected diffuse intrinsic pontine glioma Nationwide Children's Hospital Recruiting No outcome recorded
Phase 12 trials
Phase 1 NCT03056599 Dec 2016 → Jul 2021 soft tissue sarcoma Presage Biosciences Completed No outcome recorded
Phase 1 NCT02122913 May 2014 → Feb 2017 NTRK fusion positive cancer Bayer Completed No outcome recorded
Phase 1/21 trial
Phase 1/2 NCT02637687 Dec 2015 → Jul 2024 overdue NTRK fusion positive cancer Bayer Active not recruiting No outcome recorded
Phase 212 trials
Phase 2 NCT06563999 Nov 2024 → Nov 2027 expected lung carcinoma Sun Yat-sen University Recruiting No outcome recorded
Phase 2 NCT05783323 Feb 2024 → Oct 2026 expected childhood malignant neoplasm, differentiated thyroid carcinoma Children's Hospital of Philadelphia Recruiting No outcome recorded
Phase 2 NCT06145308 Aug 2023 → Jul 2028 expected salivary gland carcinoma Peking Union Medical College Recruiting No outcome recorded
Phase 2 NCT05725200 Sep 2022 → Dec 2038 expected colorectal cancer Oslo University Hospital Recruiting No outcome recorded
Phase 2 NCT06482086 Jun 2021 → Dec 2024 overdue thyroid gland carcinoma West China Hospital Recruiting No outcome recorded
Phase 2 NCT04879121 Apr 2021 → Nov 2027 expected metastatic malignant neoplasm M.D. Anderson Cancer Center Recruiting No outcome recorded
Phase 2 NCT03834961 Oct 2019 → Sep 2024 overdue acute leukemia, central nervous system neoplasm, pediatric fibrosarcoma Children's Oncology Group Active not recruiting No outcome recorded
Phase 2 NCT03213704 Aug 2017 → Sep 2024 overdue Ewing sarcoma/peripheral primitive neuroectodermal tumor, Langerhans cell histiocytosis, ependymoma, hepatoblastoma +9 National Cancer Institute (NCI) Active not recruiting No outcome recorded
Phase 2 NCT03155620 Jul 2017 → Mar 2025 overdue Ewing sarcoma, Langerhans cell histiocytosis, Wilms tumor, conventional osteosarcoma +13 National Cancer Institute (NCI) Active not recruiting No outcome recorded
Phase 2 NCT06390852 Mar 2017 → Dec 2026 expected adult lymphoma, plasma cell myeloma, refractory malignant neoplasm National Cancer Institute (NCI) Active not recruiting No outcome recorded
Phase 42 trials
Phase 4 NCT07010393 Jul 2025 → Dec 2027 expected thyroid tumor Fujian Medical University Not yet recruiting No outcome recorded
Phase 4 NCT06195228 Jan 2020 → Dec 2026 expected Fudan University Recruiting No outcome recorded
Phase not stated1 trial
NCT03025360 NTRK fusion positive cancer Bayer Approved for marketing No outcome recorded

Evidence & citations 16 cited values

Every value below carries the sentence it was read from. 21 sources stand behind the page.

FieldValueCited text
Known as Larotrectinib larotrectinib 100 mg orally twice daily” NCT06145308
18

NCT05783323

NCT02122913

NCT02465060

NCT03834961

NCT02637687

NCT03213704

NCT06482086

NCT06195228

NCT03155620

NCT06563999

NCT03025360

NCT03056599

NCT06390852

NCT04655404

NCT02576431

NCT07010393

NCT05725200

NCT04879121

Known as ARRY-470 ChEMBL registry synonym — accepted as the source's own label CHEMBL3889654
Known as ARRY-470 SULFATE ChEMBL registry synonym — accepted as the source's own label CHEMBL3889654
Known as BAY2757556 “Larotrectinib (Vitrakvi, BAY2757556)” NCT02637687
3

“Larotrectinib (Vitrakvi, BAY2757556)” NCT03025360

BAY2757556 (Larotrectinib, Vitrakvi)” NCT02576431

“Larotrectinib (Vitrakvi, BAY2757556)” NCT02122913

Known as larotrectinib sulfate “Patients receive larotrectinib sulfate orally (PO) or via nasogastric (NG)- or gastric (G)-tube twice per day (BID) on days 1-28.” NCT03213704
Known as LOXO-101 ChEMBL registry synonym — accepted as the source's own label CHEMBL3889654
Known as LOXO-101 (ARRY-470) ChEMBL registry synonym — accepted as the source's own label CHEMBL3889654
Known as LOXO-101 sulfate ChEMBL registry synonym — accepted as the source's own label CHEMBL3889654
Known as LOXO-101LOXO 101LOXO101A-9048 ChEMBL registry synonym — accepted as the source's own label CHEMBL3889654
Known as Vitrakvi “BAY2757556 (Larotrectinib, Vitrakvi)” NCT02576431
3

“Larotrectinib (Vitrakvi, BAY2757556)” NCT02122913

“Larotrectinib (Vitrakvi, BAY2757556)” NCT02637687

“Larotrectinib (Vitrakvi, BAY2757556)” NCT03025360

Action Inhibit “because it blocks the action of these genes in cancer cells” NCT02122913
Modality Small molecule “Larotrectinib, a highly selective small-molecule inhibitor of the TRK kinases” PMID 29606586 Mar 2018
Route Oral “receive larotrectinib (LOXO-101) PO BID” NCT02465060
Target NTRK1 “The drug may be used to treat cancer with a change in a particular gene (NTRK1, NTRK2 or NTRK3), because it blocks the action of these genes in cancer cells.” NCT02122913
Target NTRK2 “The drug may be used to treat cancer with a change in a particular gene (NTRK1, NTRK2 or NTRK3), because it blocks the action of these genes in cancer cells.” NCT02122913
Target NTRK3 “The drug may be used to treat cancer with a change in a particular gene (NTRK1, NTRK2 or NTRK3), because it blocks the action of these genes in cancer cells.” NCT02122913