Drug · Small Molecule

Vemurafenib

CI-DRUG-00000635Explore in graph →NCIt C64768 CHEMBL1229517 CIViC Approved

Regulatory

Approvals (9)

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-dpdRegulatory9 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 ZELBORAF (DIN 02380242) under ATC L01EC01 VEMURAFENIB. Indications are not published in the Drug Product Database — see the Health Canada Product Monograph.	approvedsource: Marketed	Mar 5, 2012	health-canada-dpd
EU EMA	Melanoma	Vemurafenib is indicated in monotherapy for the treatment of adult patients with BRAF-V600-mutation-positive unresectable or metastatic melanoma.	approved	Feb 17, 2012	ema
US FDA	—	Efficacy supplement 2017-11-06 (see label)	approved	Nov 6, 2017	openfda
US FDA	—	Efficacy supplement 2017-09-13 (see label)

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 14 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	Phase 3	Trials	Approvals	Jurisdictions	First approval	First trial
All cancers	Approved	Phase 4	18	7	11	116	9	CA, EU, US	Aug 17, 2011	2006-11
Malignant Thyroid Gland Neoplasm	Phase3	Phase 3	4	2	1	7	0	—	—	Nov 7, 2012
Non-Hodgkin Lymphoma	Phase3	Phase 3	4	2	1	8	0	—	—	Apr 12, 2012
Malignant Lung Neoplasm	Phase3	Phase 3	3	2	2	5	0

Curated evidence

Clinical evidence (261)

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

civicCuratedShowing 1–50 of 261 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
BRAF Fusion2
PLX4720 + Vemurafenib

Clinical trials

Trials with this intervention (119)

Most recently updated first, 50 per page.

clinicaltrialsPublishedShowing 1–50 of 119 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
NCT01709292	Vemurafenib Neoadjuvant Trial in Locally Advanced Thyroid Cancer	active not recruiting	Phase 2	24	M.D. Anderson Cancer CenterOTHER	1	Sep 14, 2026	clinicaltrials
NCT04302025	A Study of Multiple Therapies in Biomarker-selected Participants With Resectable Stages IB-III Non-small Cell Lung Cancer (NSCLC)(NAUTIKA1)

Molecule

Record

Kind: Small Molecule
Mechanism: Serine/threonine-protein kinase B-raf inhibitor
Targets: 1 gene
UNII: 207SMY3FQT
Aliases: 1-propanesulfonamide, N-(3-((5-(4-chlorophenyl)-1h-pyrrolo(2,3-b)pyridin-3-yl)carbonyl)-2,4-difluorophenyl)-, BRAF (V600E) Kinase Inhibitor RO5185426, PLX 4032, PLX-4032, PLX4032, RG 7204, RG7204, RO 5185426, RO-51-85426, Vemurafenib, Zelboraf

Data updated 21 hours agoSource updated unknown

Identifiers

Codes (4)

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

System	Code	Label
ATC (WHO)	L01EC01	VEMURAFENIB
DIN (Health Canada)	02380242	ZELBORAF
DPD drug code

BRAF V600E22
Vemurafenib	Childhood Pilocytic Astrocytoma	Predictive	C	Supports Sensitivity Response	3	accepted	EID3777 In a case report, a 28 month old patient with a cervicomedullary low grade glioma compatible with pilocytic astrocytoma which was resected had a second lesion consistent with a ganglioglioma 3 years l… (full text at CIViC) PMID 25524464 · del Bufalo et al., 2014 · Open in CIViC	civic
Irinotecan + Panitumumab + VemurafenibCombination	Cholangiocarcinoma	Predictive	C	Supports Sensitivity Response	3	accepted	EID5906 A 31 year old patient with metastatic cholangiocarcinoma with BRAF V600E was treated with vemurafenib, panitumumab and irinotecan triplet therapy. By 2 months, 50% reduction of tumor volume was noted,… (full text at CIViC) PMID 26687137 · Silkin et al., 2016 · Open in CIViC	civic
Vemurafenib	Clear Cell Sarcoma of Soft TissueALIAS	Predictive	C	Supports Sensitivity Response	—	submitted	EID3779 In a case report, a clear cell sarcoma patient harboring BRAF V600E mutation with distant metastases was associated with a complete response to vemurafenib monotherapy at 8 weeks of treatment. Prior t… (full text at CIViC) PMID 26286452 · Protsenko et al., 2015 · Open in CIViC	civic
〃	Conjunctival MelanomaALIAS	Predictive	C	Supports Sensitivity Response	2	submitted	EID3790 In a conjunctival malignant melanoma patient harboring a BRAF V600E mutation, BRAF V600E was associated with response to vemurafenib treatment. Prior to identification of the BRAF V600E mutation, the … (full text at CIViC) PMID 27520988 · Maleka et al., 2016 · Open in CIViC	civic
〃	Cutaneous MelanomaALIAS	Predictive	A	Supports Sensitivity Response	5	accepted	EID1409 Phase 3 randomized clinical trial comparing vemurafenib with dacarbazine in 675 patients with previously untreated, metastatic melanoma with the BRAF V600E mutation. At 6 months, overall survival was … (full text at CIViC) PMID 21639808 · Chapman et al., 2011 · Open in CIViC	civic
Dabrafenib + Trametinib + VemurafenibCombination	Digestive System Neuroendocrine TumorALIAS	Predictive	C	Supports Sensitivity Response	3	accepted	EID1430 BRAF mutations were identified in 9% of 108 cases of high-grade colorectal neuroendocrine tumors (80% V600E). Two patients were treated with a combination of BRAF and MEK inhibition and exhibited dura… (full text at CIViC) PMID 27048246 · Klempner et al., 2016 · Open in CIViC	civic
Vemurafenib	Ganglioglioma	Predictive	C	Supports Sensitivity Response	—	submitted	EID3778 A grade I refractory brainstem ganglioglioma patient harboring BRAF V600E mutation was associated with partial response to vemurafenib monotherapy. The patient experienced significant clinical improve… (full text at CIViC) PMID 26579623 · Aguilera et al., 2016 · Open in CIViC	civic
Cobimetinib + VemurafenibCombination	Ganglioglioma	Predictive	C	Supports Sensitivity Response	1	submitted	EID11310 In this case study, a 32-year-old man, previously fit and well, presented with a two-month history of vertigo, right visual disturbance, dysphagia, and ataxia. Brain MRI revealed a large mixed solid a… (full text at CIViC) PMID 31985841 · Yau et al., 2020 · Open in CIViC	civic
Vemurafenib	GlioblastomaCURATED_BROADER	Predictive	C	Supports Sensitivity Response	—	submitted	EID3771 In a case study, a pediatric grade IV glioblastoma multiforme patient harboring BRAF V600E mutation was associated with a complete response to vemurafenib monotherapy after 4 months of treatment, whic… (full text at CIViC) PMID 24725538 · Robinson et al., 2014 · Open in CIViC	civic
〃	Hairy Cell Leukemia	Predictive	B	Supports Sensitivity Response	2	accepted	EID1579 Two clinical trials evaluated the effects of vemurafenib in 54 patients with BRAF (V600E) positive hairy-cell leukemia. The overall response rate was 98% with 19/54 having a complete response and 34/… (full text at CIViC) PMID 26352686 · Tiacci et al., 2015 · Open in CIViC	civic
〃	Hairy Cell Leukemia	Predictive	B	Supports Sensitivity Response	3	submitted	EID11315 In these two phase II studies (Italy and U.S. locations), BRAF V600E-mutant hairy-cell leukemia was treated with vemurafenib at a dose of 960 mg twice daily to investigate its efficacy. The overall re… (full text at CIViC) PMID 26352686 · Tiacci et al., 2015 · Open in CIViC	civic
〃	Hairy Cell Leukemia	Predictive	C	Supports Sensitivity Response	—	submitted	EID3766 In a refractory hairy cell leukemia patient harboring BRAF V600E mutation, BRAF V600E was associated with sensitivity to vemurafenib treatment. The patient was initially treated with conventional trea… (full text at CIViC) PMID 22621641 · Dietrich et al., 2012 · Open in CIViC	civic
〃	Hairy Cell Leukemia	Predictive	C	Supports Sensitivity Response	—	submitted	EID3767 In a case report, a hairy cell leukemia (HCL) patient harboring BRAF V600E mutation was associated with a complete response to vemurafenib monotherapy after 3 months of treatment, which was sustained … (full text at CIViC) PMID 23300174 · Peyrade et al., 2013 · Open in CIViC	civic
〃	Hairy Cell Leukemia	Predictive	C	Supports Sensitivity Response	—	submitted	EID3768 In a hairy cell leukemia patient harboring BRAF V600E mutation, BRAF V600E mutation was associated with improved response to vemurafenib treatment. The patient was treated with 3 lines of chemotherapy… (full text at CIViC) PMID 23278307 · Follows et al., 2013 · Open in CIViC	civic
〃	Hairy Cell Leukemia	Predictive	C	Supports Sensitivity Response	—	submitted	EID3769 As a follow-up to a previous study (23300174), a hairy cell leukemia patient harboring BRAF V600E mutation was associated with response to vemurafenib monotherapy. The patient was previously treated w… (full text at CIViC) PMID 25815361 · Bailleux et al., 2015 · Open in CIViC	civic
〃	Hairy Cell Leukemia	Predictive	C	Supports Sensitivity Response	—	submitted	EID3770 A hairy cell leukemia patient with extensive CNS involvement patient harboring BRAF V600E mutation was associated with complete response to vemurafenib monotherapy. Upon identification of the BRAF V60… (full text at CIViC) PMID 27116997 · McDowell et al., 2016 · Open in CIViC	civic
〃	High-Grade Glioma, NOS	Predictive	B	Supports Sensitivity Response	—	rejected	EID3763 A study of pediatric malignant astrocytomas found that tumors with BRAF V600E mutations also had homozygous deletions of CDKN2A more frequently than those with wildtype BRAF (71% vs. 8% , P=0.0016, F… (full text at CIViC) PMID 20068183 · Schiffman et al., 2010 · Open in CIViC	civic
〃	High-Grade Glioma, NOS	Predictive	B	Supports Sensitivity Response	—	rejected	EID3764 In a study of pilocytic astrocytomas, presence of the BRAF V600E mutation was more frequently detected in extra-cerebellar pilocytic astrocytomas than cerebellar ones (20% vs. 2% , P=0.009, Fisher�s… (full text at CIViC) PMID 21274720 · Schindler et al., 2011 · Open in CIViC	civic
〃	High-Grade Glioma, NOS	Predictive	B	Supports Sensitivity Response	—	rejected	EID3765 In a study of low grade pediatric gliomas, 19 of 157 tumors had a BRAF V600E mutation. Tumors located in the cerebrum had a higher frequency of BRAF mutations than those in the cerebellum or midline (… (full text at CIViC) PMID 22492957 · Horbinski et al., 2012 · Open in CIViC	civic
〃	High-Grade Glioma, NOS	Predictive	C	Supports Sensitivity Response	2	submitted	EID12719 In an open-label phase II Pediatric MATCH subprotocol of vemurafenib for relapsed or refractory solid tumors with BRAF V600 mutations (NCT03220035; n = 4 evaluable), all four treated patients carried … (full text at CIViC) PMID 38873934 · Nelson et al., 2024 · Open in CIViC	civic
〃	High-Grade Glioma, NOS	Predictive	D	Supports Sensitivity Response	—	submitted	EID3762 In a mouse xenograft model, tumors derived from the glioblastoma cell line AM-38 that were treated with vemurafenib had reduced tumor growth and improved survival compared to control treated mice (P=0… (full text at CIViC) PMID 22038996 · Nicolaides et al., 2011 · Open in CIViC	civic
〃	Histiocytoma	Predictive	C	Supports Sensitivity Response	—	submitted	EID3789 A primary central nervous system (CNS)-histiocytic sarcoma patient harboring BRAF V600E mutation was associated with improved response to vemurafenib treatment. The patient was treated with vemurafeni… (full text at CIViC) PMID 25209580 · Idbaih et al., 2014 · Open in CIViC	civic

BRAF V6004
Vemurafenib	Cholangiocarcinoma	Predictive	B	Supports Sensitivity Response	4	accepted	EID5905 Eight patients with BRAF V600 (7 with V600E, 1 with V600 unknown) mutated cholangiocarcinoma received vemurafenib. Only one patients showed partial response and duration of response of this patient wa… (full text at CIViC) PMID 26287849 · Hyman et al., 2015 · Open in CIViC	civic
〃	Erdheim-Chester Disease	Predictive	A	Supports Sensitivity Response	5	submitted	EID11237 In this histology-independent, phase 2, open-label study, 26 patients (22 with Erdheim-Chester disease (ECD) and 4 Langerhans cell histiocytosis (LCH)) were administered 960 mg, twice daily doses of v… (full text at CIViC) PMID 29188284 · Diamond et al., 2018 · Open in CIViC	civic
〃	Langerhans Cell Histiocytosis	Predictive	B	Supports Sensitivity Response	3	accepted	EID1575 Patients with BRAF-V600 mutated cancers were identified (n=122) and clinical response to vemurafenib was evaluated. Of the 14 patients with Langerhans’-cell histiocytosis, 43% of patients had a respo… (full text at CIViC) PMID 26287849 · Hyman et al., 2015 · Open in CIViC	civic
〃	Langerhans Cell Histiocytosis	Predictive	B	Supports Sensitivity Response	4	submitted	EID11302 In this open-label, non-randomized trial, 26 patients with BRAF V600–mutant Erdheim-Chester Disease or Langerhans Cell Histiocytosis (22 with Erdheim-Chester Disease, 4 with Langerhans Cell Histiocyto… (full text at CIViC) PMID 29188284 · Diamond et al., 2018 · Open in CIViC	civic

BRAF V600R2
Vemurafenib	Cutaneous MelanomaALIAS	Predictive	C	Supports Sensitivity Response	3	submitted	EID4172 An 81 year old male was diagnosed with ulcerated cutaneous melanoma of the occiput (M1c), and had undergone several local resections and radiotherapy. A metastasis was found to harbor BRAF V600R mutat… (full text at CIViC) PMID 23237741 · Klein et al., 2013 · Open in CIViC	civic
Dabrafenib + VemurafenibSubstitutes	Cutaneous MelanomaALIAS	Predictive	B	Does Not Support Resistance	2	submitted	EID8948 Nine patients with BRAF V600R mutated metastatic melanoma were given dabrafenib (n=7) or vemurafenib (n=2) monotherapy as part of a compassionate access program. Five out of six radiographically asses… (full text at CIViC) PMID 23237741 · Klein et al., 2013 · Open in CIViC	civic

MET Overexpression2
Vemurafenib	Cutaneous MelanomaALIAS	Predictive	B	Supports Resistance	4	accepted	EID1581 Whole exome sequencing, transcriptome and methylome alterations in 48 melanoma samples with acquired resistance to BRAFi +/- MEKi therapy compared to patient-matched baseline melanoma tissues were ana… (full text at CIViC) PMID 26359985 · Hugo et al., 2015 · Open in CIViC	civic
〃	Cutaneous MelanomaALIAS	Predictive	B	Does Not Support Resistance	3	accepted	EID1584 FFPE tumours from patients enrolled in the BRIM2 (n = 59) and BRIM3 (n = 150) trials of vemurafenib in advanced BRAF mutant (V600E/K) melanoma were analyzed for MET expression (IHC). Pretreatment MET … (full text at CIViC) PMID 23802768 · Jubb et al., 2013 · Open in CIViC	civic

NF1 Mutation2
Vemurafenib	Cutaneous MelanomaALIAS	Predictive	C	Supports Resistance	4	accepted	EID1470 NF1 was identified in an unbiased RNAi screen as mediator of resistance to BRAF inhibition in BRAF mutant melanoma. Knockdown of NF1 led to PLX4720 resistance in-vitro. MEK inhibition with AZD6244 (se… (full text at CIViC) PMID 23288408 · Whittaker et al., 2013 · Open in CIViC	civic
〃	Cutaneous MelanomaALIAS	Predictive	D	Supports Resistance	2	accepted	EID1468 Preclinical study of mouse melanoma in-vitro and in-vivo. In-vitro treament of Braf-mutant melanomas were 10-fold more sensitive to vermurafenib (PLX4032) treatment than Braf/Nf1-mutant melanomas. In … (full text at CIViC) PMID 23171796 · Maertens et al., 2013 · Open in CIViC	civic