Drug · Small Molecule

Larotrectinib

CI-DRUG-00000331Explore in graph →NCIt C115977 CHEMBL3889654 CIViC Approved

Regulatory

Approvals (10)

Each record names the authority, jurisdiction, indication text and status. A drug approved in one jurisdiction for one indication is not 'approved' in general.

health-canada-dpdRegulatory10 approval records from 3 sources (hover a row badge for its dataset) · authority, jurisdiction and indication text as published.

Jurisdiction · authority	Cancer	Indication	Status	Approval date	Source
CA Health Canada	—	Marketed in Canada as VITRAKVI (DIN 02490315) under ATC L01EX12 LAROTRECTINIB. Indications are not published in the Drug Product Database — see the Health Canada Product Monograph.	approvedsource: Marketed	Nov 13, 2020	health-canada-dpd
CA

Health Canada records are DIN-level: one row per marketed product (brand, strength, form). The Drug Product Database does not publish indications, so no cancer is stated for these rows, and a cancelled or dormant DIN is the status of that one product — not a withdrawal of the molecule.

Data updated 2 days agoSource updated unknown

Derived

Development pipeline

Most advanced stage across all cancers, then per top-level cancer reached through trial conditions or approval indications. Approval in any ingested jurisdiction outranks trial phase; counts are interventional studies.

Scope	Stage	Max phase	Active	Recruiting	Trials	Approvals	Jurisdictions	First approval	First trial
All cancers	Approved	Phase 4	13	5	15	10	CA, EU, US	Nov 26, 2018	May 4, 2014
Non-Hodgkin Lymphoma	Phase2	Phase 2	4	0	4	0	—	—	Aug 17, 2015
Malignant Central Nervous System Neoplasm	Phase2	Phase 2	3	1	3	0	—	—	Jul 31, 2017
Malignant Liver Neoplasm	Phase2	Phase 2	3	0	3	0

Curated evidence

Clinical evidence (79)

CIViC items in which this therapy appears, grouped by cancer context, then molecular profile. 50 items per page.

civicCuratedShowing 1–50 of 79 evidence items · levels, directions and significance as curated at the source; each row links to its CIViC record.

Therapy	Cancer	Type	Level	Direction · significance	Rating (1–5)	Status	Evidence	Source
NTRK2 Fusion1
Larotrectinib	Acute Myeloid Leukemia

Clinical trials

Trials with this intervention (20)

Most recently updated first, 50 per page.

clinicaltrialsPublishedShowing 1–20 of 20 studies · status and phase as posted by the registrant; each row links to the study record.

NCT	Title	Status	Phase	Enrollment (n)	Sponsor	Countries	Last update	Source
NCT07843550	A Phase 1/2 Trial Of Larotrectinib And Pembrolizumab In Patients With Solid Tumors Previously Treated With Immune Checkpoint Inhibitor Monotherapy	not yet recruiting	Phase 1 / Phase 2	22	M.D. Anderson Cancer CenterOTHER	1	Sep 28, 2026	clinicaltrials
NCT06390852	Testing LOXO-101 as Potentially Targeted Treatment in Cancers With NTRK Genetic Changes (MATCH - Subprotocol Z1E)

Molecule

Record

Kind: Small Molecule
Mechanism: Neurotrophic tyrosine kinase receptor inhibitor
Targets: 3 genes
UNII: PF9462I9HX
Aliases: 1-Pyrrolidinecarboxamide, N-(5-((2R)-2-(2,5-difluorophenyl)-1-pyrrolidinyl)pyrazolo(1,5-a)pyrimidin-3-yl)-3-hydroxy-, (3S)-, 3S)-N-(5-((2R)-2-(2,5-Difluorophenyl)pyrrolidin-1-yl)pyrazolo(1,5-a)pyrimidin-3-yl)-3-hydroxypyrrolidine- 1-carboxamide, ARRY 470, ARRY-470, BAY-2757556, BAY2757556, Larotrectinib, Larotrectinib Sulfate, LOXO 101, LOXO-101, LOXO101, LOXO-101 (ARRY-470), LOXO-101LOXO 101LOXO101A-9048, VITRAKVI

Data updated 21 hours agoSource updated unknown

Identifiers

Codes (9)

Upstream identifiers kept as first-class codes; each links to its registry where a stable public URL exists.

System	Code	Label
ATC (WHO)	L01EX12	LAROTRECTINIB
ATC (WHO)	L01XE53

NTRK2 Fusion2
Larotrectinib	Anaplastic Ependymoma	Predictive	C	Supports Sensitivity Response	4	accepted	EID10360 An MRI in a previously healthy 6-year-old boy now presenting with neurologic abnormalities and seizures revealed a mass obstructing the foramen of Monro and diffuse leptomeningeal dissemination. The p… (full text at CIViC) PMID 34651095 · Mangum et al., 2021 · Open in CIViC	civic
〃	GlioblastomaCURATED_BROADER	Predictive	C	Supports Sensitivity Response	3	accepted	EID10361 An MRI of 26-year-old man presenting with motor weakness and language difficultly revealed a lesion in the left frontal lobe that extended across the corpus callosum. Histologic analysis of the biopsy… (full text at CIViC) PMID 34041811 · Shepherd et al., 2021 · Open in CIViC	civic

NTRK3 Fusion12
Larotrectinib	B Lymphoblastic Leukemia/LymphomaCURATED_BROADER	Predictive	C	Supports Sensitivity Response	3	accepted	EID7993 Case report of a 61-year-old man with B-ALL. An initial analysis detected the ETV6-NTRK3 fusion and a NRASGly12Asp mutation. Initial therapies included chemotherapy and CAR-T infusion. Relapse 6 weeks… (full text at CIViC) PMID 31905241 · Nardi et al., 2020 · Open in CIViC	civic
〃	B Lymphoblastic Leukemia/LymphomaCURATED_BROADER	Predictive	D	Supports Sensitivity Response	4	accepted	EID6930 Both in vivo and in vitro models demonstrated Larotrectinib was an effective treatment against cells harboring ETV6-NTRK3. In YFP-luciferase PDX mice from a 16-year-old with Ph-like B-ALL, flow cytome… (full text at CIViC) PMID 29880614 · Roberts et al., 2018 · Open in CIViC	civic
〃	B Lymphoblastic Leukemia/LymphomaCURATED_BROADER	Predictive	D	Supports Sensitivity Response	3	accepted	EID8930 Conventional cytogenetics, FISH, and an Abbott break-apart probe of a peripheral blood sample collected at diagnosis of a 6-year-old boy with B-cell ALL and CNS infiltration (National Cancer Institute… (full text at CIViC) PMID 31725893 · Schewe et al., 2019 · Open in CIViC	civic
Etoposide + Larotrectinib + MethotrexateCombination	B Lymphoblastic Leukemia/LymphomaCURATED_BROADER	Predictive	C	Supports Sensitivity Response	4	accepted	EID8917 All lines of conventional therapy (including allogeneic HSCT, blinatumomab, and CAR T cells) were inefficient for a 6-year-old boy with B-cell ALL and CNS infiltration (National Cancer Institute high … (full text at CIViC) PMID 31725893 · Schewe et al., 2019 · Open in CIViC	civic
Larotrectinib	Breast Secretory Carcinoma	Predictive	C	Supports Sensitivity Response	3	accepted	EID6475 Shukla et al report on a remarkable case study of a 14 year old pediatric patient with refractory secretory breast carcinoma first presenting as a fibroadenoma at 8 yo. Over the next years, she experi… (full text at CIViC) PMID 29623306 · Shukla et al., 2017 · Open in CIViC	civic
〃	Congenital Mesoblastic Nephroma	Predictive	C	Supports Sensitivity Response	4	accepted	EID10840 A 23 month old (at the time of report) male was diagnosed prenatally with Stage II cellular mesoblastic nephroma (MN) on the right kidney, which was surgically removed on the sixth day of life. ETV6::… (full text at CIViC) PMID 29893456 · Halalsheh et al., 2018 · Open in CIViC	civic
〃	Hematopoietic and Lymphoid Cell NeoplasmALIAS	Predictive	C	Supports Sensitivity Response	3	submitted	EID6395 Rearrangements involving the neurotrophic receptor kinase genes (NTRK1, NTRK2, and NTRK3; hereafter referred to as TRK) produce oncogenic fusions in a wide variety of cancers in adults and children. I… (full text at CIViC) PMID 29920189 · Taylor et al., 2018 · Open in CIViC	civic
〃	High-Grade Glioma, NOS	Predictive	C	Supports Sensitivity Response	3	submitted	EID10931 A 3 year old patient was initially diagnosed with a high grade glioma at 5 months of age. She was treated with 13 cycles of chemotherapy. 4 months after completing initial treatment, she showed progre… (full text at CIViC) PMID 30220707 · Ziegler et al., 2018 · Open in CIViC	civic
〃	Infantile FibrosarcomaALIAS	Predictive	A	Supports Sensitivity Response	4	accepted	EID6099 Four out of six patients with infantile fibrosarcoma and a ETV6-NTRK3 fusion had a partial response, the remaining two had a complete response with larotrectinib. Treatment was done within a multicent… (full text at CIViC) PMID 29606586 · Laetsch et al., 2018 · Open in CIViC	civic
〃	Infantile FibrosarcomaALIAS	Predictive	C	Supports Sensitivity Response	2	accepted	EID6473 Nagasubramanian et al reports on a case of refractory infantile fibrosarcoma (IFS) with constitutive activation of the tropomyosin‐related kinase (TRK) signaling pathway from an ETS variant gene 6–neu… (full text at CIViC) PMID 27093299 · Nagasubramanian et al., 2016 · Open in CIViC	civic
〃	Infantile FibrosarcomaALIAS	Predictive	C	Supports Sensitivity Response	3	accepted	EID7418 In a pediatric phase 1 trial (NCT02637687) after initial chemotherapy failure, two ETV6-NTRK3 positive children (2 year old and 5 month old) were treated with Larotrectinib, resulting in reduced tumor… (full text at CIViC) PMID 30204247 · DuBois et al., 2018 · Open in CIViC	civic
〃	Infantile FibrosarcomaALIAS	Predictive	C	Supports Sensitivity Response	3	accepted	EID10717 This case report describes a male infant with a recurrent (post-resection) infantile fibrosarcoma (IFS) with lymph node involvement and ETV6-NTRK3 fusion. The tumor did not respond to chemotherapy but… (full text at CIViC) PMID 32223937 · Bielack et al., 2019 · Open in CIViC	civic

NTRK1 Fusion5
Larotrectinib	Cholangiocarcinoma	Predictive	C	Supports Sensitivity Response	2	submitted	EID6052 In a Phase I/II study of Larotrectinib, highly selective TRK inhibitor, two patient with cholangiocarcinoma with NTRK fusion was enrolled. One of the two patient showed PR. PMID 29466156 · Drilon et al., 2018 · Open in CIViC	civic
Crizotinib + Larotrectinib + LestaurtinibSubstitutes	Lung Adenocarcinoma	Predictive	D	Supports Sensitivity Response	3	accepted	EID1019 Two patients were found to harbor gene fusions maintaining the kinase domain of NTRK1 (3' partner), specifically: MPRIP-NTRK1 and CD74-NTRK1. Both of these fusions lead to constitutive kinase activit… (full text at CIViC) PMID 24162815 · Vaishnavi et al., 2013 · Open in CIViC	civic
Larotrectinib	Malignant NeoplasmALIAS	Predictive	C	Supports Sensitivity Response	2	accepted	EID2953 Results from a phase I study show that the TRK inhibitor LOXO-101 is well tolerated and effective, with patients whose tumors bear NTRK fusions responding well and durably to this targeted therapy. Th… (full text at CIViC) PMID 26603524 · 2016 · Open in CIViC	civic
〃	Malignant Pancreatic Neoplasm	Predictive	C	Supports Sensitivity Response	2	submitted	EID6051 In a Phase I/II study of Larotrectinib, highly selective TRK inhibitor, one patient with pancreatic cancer with NTRK fusion showed PR. PMID 29466156 · Drilon et al., 2018 · Open in CIViC	civic
〃	Malignant Pancreatic Neoplasm	Predictive	C	Supports Sensitivity Response	3	submitted	EID7649 A patient with pancreatic cancer harboring NTRK fusion was treated with larotrectinib. The best overall response was PR at 2 months but the tumor progressed after 6 months. PMID 31605106 · O'Reilly et al., 2019 · Open in CIViC	civic

NTRK1 Fusion4
Larotrectinib	Gliosarcoma	Predictive	C	Supports Sensitivity Response	4	accepted	EID11746 A 9-month-old boy with no family history of cancer presented with increased head circumference, and MRI demonstrated a solid lesion in the left frontal lobe. A subtotal surgical resection was performe… (full text at CIViC) PMID 35034013 · Mançano et al., 2022 · Open in CIViC	civic
〃	Lung Non-Small Cell CarcinomaCURATED_BROADER	Predictive	C	Supports Sensitivity Response	3	accepted	EID11744 In the clinical trial NCT01775072, one patient had TPR::NTRK1 positive non-small cell lung cancer, and had a partial response with larotrectinib treatment before eventual progression. PMID 33004339 · Cocco et al., 2021 · Open in CIViC	civic
〃	Malignant Colorectal NeoplasmCURATED_BROADER	Predictive	C	Supports Sensitivity Response	4	accepted	EID11706 A female patient, 43 presented with right lower quadrant pain, and was surgically treated for perforated appendicitis including segmental small-bowel resection. Invasive adenocarcinoma stage IIB (pT4a… (full text at CIViC) PMID 35815302 · Kasi et al., 2022 · Open in CIViC	civic
〃	Malignant Thyroid Gland Neoplasm	Predictive	C	Supports Sensitivity Response	4	accepted	EID11748 A 4.3-year-old girl had I-131–refractory papillary thyroid carcinoma (PTC). There was lymph node involvement and lung metastases. She underwent thyroidectomy and neck dissection, and administration of… (full text at CIViC) PMID 34237031 · Lee et al., 2021 · Open in CIViC	civic

NTRK1 Fusion3
Larotrectinib	Infantile FibrosarcomaALIAS	Predictive	C	Supports Sensitivity Response	3	accepted	EID6103 One patient with infantile fibrosarcoma and a NTRK1-SQSTM1 fusion was treated with larotrectinib and exhibited a partial response. Treatment was done within a multicentre, open-label, phase 1/2 study … (full text at CIViC) PMID 29606586 · Laetsch et al., 2018 · Open in CIViC	civic
〃	Infantile FibrosarcomaALIAS	Predictive	C	Supports Sensitivity Response	3	accepted	EID8865 In a pediatric phase 1 trial (NCT02637687), a 2 year old with infantile fibrosarcoma of the shoulder harbored the SQSTM1-NTRK1 fusion. After two surgical resections and multiple rounds of chemotherapy… (full text at CIViC) PMID 30204247 · DuBois et al., 2018 · Open in CIViC	civic
〃	Lung Adenocarcinoma	Predictive	C	Supports Sensitivity Response	3	accepted	EID11574 Drilon et al (2022) assessed the efficacy and safety of larotrectinib in lung cancer patients with TRK fusions in a phase II adult/young adult trial (NCT02576431) and a phase I adult trial (NCT0212291… (full text at CIViC) PMID 35085007 · Drilon et al., 2022 · Open in CIViC	civic

NTRK1 Fusion2
Larotrectinib	Lung Carcinoma	Predictive	C	Supports Sensitivity Response	3	accepted	EID11692 The goal of this study was to assess the efficacy and safety of larotrectinib in patients with TRK fusion–positive lung cancers. This was achieved by reviewing data from two global, multicenter clinic… (full text at CIViC) PMID 35085007 · Drilon et al., 2022 · Open in CIViC	civic
Larotrectinib + LestaurtinibSubstitutes	Malignant Colorectal NeoplasmCURATED_BROADER	Predictive	D	Supports Sensitivity Response	2	accepted	EID978 NTRK1 fusions (one LMNA-NTRK1 and two TPM3-NTRK1) were identified in 2% of Korean colorectal cancer patients (147 patients). TrkA (NTRK1) protein expression was associated with presence of NTRK1 gene … (full text at CIViC) PMID 26716414 · Park et al., 2016 · Open in CIViC	civic