Definition and research guide
This page combines a MeSH definition, publication statistics and selected research abstracts. It is a starting point for reading the literature, not a medical textbook or a systematic evidence review.
Content available: 4 complete abstracts (968 words), 10 references. Full article text is not stored or reproduced here; follow the PMID, DOI or PMC links to read the original sources and their access terms.
Definition
An indole sulfonamide compound and inhibitor of braf kinases that is used for the treatment of unresectable or metastatic melanoma.
Definition: NLM Medical Subject Headings (MeSH) scope note (Public domain (U.S. National Library of Medicine); attribution requested). View the MeSH record.
Also known as: PLX4032; PLX 4032; Zelboraf; R05185426; RG7204; RG-7204; RG 7204.
Where it fits in the MeSH classification
- Broader topics
- Sulfonamides; Indoles
Related concepts
- Braf Kinases
- Melanoma
Research landscape
PubMed indexes 1,809 records tagged with the MeSH heading “Vemurafenib” (counts retrieved 2026-10-31). 484 of them (26.8%) were published from 2020 onward, and the busiest period so far is 2010-2019 with 1,342 records.
Reviews account for 228 records (12.6%) and meta-analyses for 11. Randomized controlled trials number 44 (2.4%) as a share of all records on the topic.
Show the data
| Period | Records |
|---|---|
| through 1999 | 0 |
| 2000-2009 | 0 |
| 2010-2019 | 1,342 |
| 2020 onward | 484 |
Publication-type chart data
| Type | Records | Share |
|---|---|---|
| Reviews | 228 | 12.6% |
| Meta-analyses | 11 | 0.6% |
| Randomized controlled trials | 44 | 2.4% |
Method: PubMed query "Vemurafenib"[MeSH Terms] with publication-type and date filters, retrieved 2026-10-31.
Reading the evidence in drugs and chemicals
Studies of a chemical or drug can answer very different questions: what it is and how it behaves in cells and animals, how the body handles it, and whether it changes outcomes in people. Laboratory and animal findings often do not carry over to patients, and observational studies of exposures are confounded by who uses a substance and why. Nothing on this page is a recommendation to use, avoid, start or stop any substance or medicine; talk to a qualified clinician or pharmacist about your own situation. Check the dose, the species, the comparison group and whether a patient-relevant outcome was measured.
Evidence from open-access studies
These open-access studies come from PubMed’s best-match results for this heading (papers whose main topic it is), ranked here by citation count, with one recent addition. Each abstract is reproduced in full under its recorded open licence so that you can read the authors’ own words. They illustrate the literature; they are not a systematic summary of it. Citation counts measure attention, not reliability or clinical benefit.
| Study / authors | Year | Source label / design | Europe PMC citations | Licence / source |
|---|---|---|---|---|
| Mutant-selective degradation by BRAF-targeting PROTACs | 2021 | Research article Design not recorded in structured metadata | 128 | cc by PMID 33568647 · DOI · PMC7876048 |
| Tissue-engineered 3D melanoma model with blood and lymphatic capillaries for drug development | 2018 | Research article Design not recorded in structured metadata | 69 | cc by PMID 30181613 · DOI · PMC6123405 |
| Reprogramming miRNAs global expression orchestrates development of drug resistance in BRAF mutated melanoma | 2019 | Research article Design not recorded in structured metadata | 58 | cc by PMID 30254376 · DOI · PMC6748102 |
| Quaternary Phosphonium Salts Outperformed Vemurafenib (PLX) and Etoposide Against BRAFV600D,V600E PLX-Resistant Melanoma and MDR Neuroblastoma, Exhibiting No/Low Toxicity on 3T3/HaCaT Cells | 2026 | Research article Design not recorded in structured metadata | 2 | cc by PMID 41977352 · DOI · PMC13072898 |
Mutant-selective degradation by BRAF-targeting PROTACs [1]
Abstract reading summary — source excerpts
- Opening excerpt
- Over 300 BRAF missense mutations have been identified in patients, yet currently approved drugs target V600 mutants alone.
- Closing excerpt
- This study highlights the degree of selectivity achievable with degradation-based approaches by targeting mutant BRAF-driven cancers while sparing BRAFWT, providing an anti-tumor drug modality that expands the therapeutic window.
- Interpretation limits
- Verbatim excerpts from the authors’ abstract; the closing excerpt may describe a finding, limitation or recommendation. Design, population, effect size and uncertainty have not been independently extracted or appraised. Read the complete abstract and linked paper before interpreting the findings.
Read complete abstract (180 words)
Over 300 BRAF missense mutations have been identified in patients, yet currently approved drugs target V600 mutants alone. Moreover, acquired resistance inevitably emerges, primarily due to RAF lesions that prevent inhibition of BRAF V600 with current treatments. Therefore, there is a need for new therapies that target other mechanisms of activated BRAF. In this study, we use the Proteolysis Targeting Chimera (PROTAC) technology, which promotes ubiquitination and degradation of neo-substrates, to address the limitations of BRAF inhibitor-based therapies. Using vemurafenib-based PROTACs, we achieve low nanomolar degradation of all classes of BRAF mutants, but spare degradation of WT RAF family members. Our lead PROTAC outperforms vemurafenib in inhibiting cancer cell growth and shows in vivo efficacy in a Class 2 BRAF xenograft model. Mechanistic studies reveal that BRAFWT is spared due to weak ternary complex formation in cells owing to its quiescent inactivated conformation, and activation of BRAFWT sensitizes it to degradation. This study highlights the degree of selectivity achievable with degradation-based approaches by targeting mutant BRAF-driven cancers while sparing BRAFWT, providing an anti-tumor drug modality that expands the therapeutic window.
Abstract reproduced from the open-access article under the recorded cc by licence; © the authors. PMID 33568647 · DOI · PMC7876048
Tissue-engineered 3D melanoma model with blood and lymphatic capillaries for drug development [2]
Abstract reading summary — source excerpts
- Opening excerpt
- While being the rarest skin cancer, melanoma is also the deadliest.
- Closing excerpt
- Chronic treatment with vemurafenib was applied to the model and elicited a dose-dependent response on proliferation and apoptosis, making it a promising tool to test new compounds in a human-like environment.
- Interpretation limits
- Verbatim excerpts from the authors’ abstract; the closing excerpt may describe a finding, limitation or recommendation. Design, population, effect size and uncertainty have not been independently extracted or appraised. Read the complete abstract and linked paper before interpreting the findings.
Read complete abstract (156 words)
While being the rarest skin cancer, melanoma is also the deadliest. To further drug discovery and improve clinical translation, new human cell-based in vitro models are needed. Our work strives to mimic the melanoma microenvironment in vitro as an alternative to animal testing. We used the self-assembly method to produce a 3D human melanoma model exempt of exogenous biomaterial. This model is based on primary human skin cells and melanoma cell lines while including a key feature for tumor progression: blood and lymphatic capillaries. Major components of the tumor microenvironment such as capillaries, human extracellular matrix, a stratified epidermis (involucrin, filaggrin) and basement membrane (laminin 332) are recapitulated in vitro. We demonstrate the persistence of CD31+ blood and podoplanin+/LYVE-1+ lymphatic capillaries in the engineered tissue. Chronic treatment with vemurafenib was applied to the model and elicited a dose-dependent response on proliferation and apoptosis, making it a promising tool to test new compounds in a human-like environment.
Abstract reproduced from the open-access article under the recorded cc by licence; © the authors. PMID 30181613 · DOI · PMC6123405
Reprogramming miRNAs global expression orchestrates development of drug resistance in BRAF mutated melanoma [3]
Abstract reading summary — source excerpts
- Opening excerpt
- Drug resistance imposes severe limitations to the efficacy of targeted therapy in BRAF-mutated metastatic melanoma.
- Closing excerpt
- Targeting these alterations blunts the development of drug resistance.
- Interpretation limits
- Verbatim excerpts from the authors’ abstract; the closing excerpt may describe a finding, limitation or recommendation. Design, population, effect size and uncertainty have not been independently extracted or appraised. Read the complete abstract and linked paper before interpreting the findings.
Read complete abstract (145 words)
Drug resistance imposes severe limitations to the efficacy of targeted therapy in BRAF-mutated metastatic melanoma. Although this issue has been mitigated by the development of combination therapies with BRAF plus MEK inhibitors, drug resistance inevitably occurs with time and results in clinical recurrences and untreatable disease. Hence, there is strong need of developing new combination therapies and non-invasive diagnostics for the early identification of drug-resistant patients. We report here that the development of drug resistance to BRAFi is dominated by a dynamic deregulation of a large population of miRNAs, leading to the alteration of cell intrinsic proliferation and survival pathways, as well as of proinflammatory and proangiogenic cues, where a prominent role is played by the miR-199b-5p/VEGF axis. Significant alterations of miRNA expression levels are detectable in tumor biopsies and plasma from patients after disease recurrence. Targeting these alterations blunts the development of drug resistance.
Abstract reproduced from the open-access article under the recorded cc by licence; © the authors. PMID 30254376 · DOI · PMC6748102
Quaternary Phosphonium Salts Outperformed Vemurafenib (PLX) and Etoposide Against BRAFV600D,V600E PLX-Resistant Melanoma and MDR Neuroblastoma, Exhibiting No/Low Toxicity on 3T3/HaCaT Cells [4]
Abstract reading summary — source excerpts
- Opening excerpt
- Late-stage metastatic cutaneous melanoma (MCM) and neuroblastoma (NB) are the most aggressive skin and childhood cancers with survival rates of 1) and diphenyl (3 and 4) phosphonium salts were synthesized, treating commercial triphenyl phosphine and synthesizing 11-diphenylphosphanyl-undecan-1-ol (2), respectively, with benzyl bromide.
- Closing excerpt
- All compounds have the potential to be developed as adjuvants in already existing anticancer cocktails to treat MCM and/or NB.
- Interpretation limits
- Verbatim excerpts from the authors’ abstract; the closing excerpt may describe a finding, limitation or recommendation. Design, population, effect size and uncertainty have not been independently extracted or appraised. Read the complete abstract and linked paper before interpreting the findings.
Read complete abstract (487 words)
Late-stage metastatic cutaneous melanoma (MCM) and neuroblastoma (NB) are the most aggressive skin and childhood cancers with survival rates of 1) and diphenyl (3 and 4) phosphonium salts were synthesized, treating commercial triphenyl phosphine and synthesizing 11-diphenylphosphanyl-undecan-1-ol (2), respectively, with benzyl bromide. Upon full characterization, they were tested, for the first time, on MeTRAV (BRAFV600D) and MeOV (BRAFV600E) vemurafenib (PLX)-resistant MCM cells, etoposide (ETO)-sensitive (HTLA 230) and multidrug resistant (MDR) (HTLA ER) NB cells, non-tumorigenic human keratinocytes (HaCaT), and mouse embryonic fibroblasts (3T3), as well as red blood cells (RBCs). Viability of MeTRAV cells was decreased to 44.8% by administration of 1 (100 µM), in intermediate-time (48 h) treatments, while short-time exposure (24 h) to 3 (≥75 µM) and 4 (≥50 µM) was sufficient to reduce their viability to 33.6 and 32.2%. Viability of MeOV was decreased under 50% with 5 µM concentrations of 1 and 25 µM of 3 and 4, While cells were exterminated (26.9, 20.6, and 21.8%) with higher concentrations after 48 h exposure. Collectively, 1 was the better performing compound (IC50 = 6.4 µM, 48 h). Viability of HTLA ER cells was decreased under 50% upon 72 h administrations of 1 at concentrations ≥ 50 µM, 48 h (≥75 µM) and 72 h (≥50 µM) of 3, and after 72 h (≥75 µM) of 4, but 72 h exposure and high concentrations of all compounds were necessary for their extermination (31.2, 28.7, and 29.7%). Viability of HTLA 230 cells was not 1 and 4 were administered for only 24 h, while their viability was 3 at all times of exposure. At high concentrations, all compounds exterminated cells (33.6, 25.3%, 1, 48-72 h; 38.6, 30.2, and 24.7%, 3, 24-72 h; 33.2%, 4, 72 h). The best-performing compounds were 1 (IC50 = 4.0 µM, HTLA 230) and 3 (IC50 = 27.8 µM, HTLA ER) at 72 h exposure. The cytotoxic effects of compound 4 on MeTRAV cells, when exposed to 24/48 h treatments, were comparable to those of PLX on the same cells in 72 h treatments. Compound 1, in shorter 48 h treatments of PLX-R MeOV, was 2.5-fold more cytotoxic than PLX in 72 h ones. All compounds were not cytotoxic to 3T3 cells at all times of exposure; they had low cytotoxicity to HaCaT cells in 24 and 48 h treatments and were slightly cytotoxic to RBCs in 24 h ones. Compound 1 could be a promising platform to develop new intermediate-time therapies for PLX-R MeOV cells, while 4 could be used to develop 24 and 48 h treatments for PLX-R MeTRAV cells. Also, all compounds could be developed as new treatment options for both ETO-sensitive and MDR late-stage HR-NB cells, being even more effective than ETO by 1.2, 2.0, and 1.3 times (HTLA 230) and 3.2, 4.7, and 3.2 times (HTLA ER). All compounds have the potential to be developed as adjuvants in already existing anticancer cocktails to treat MCM and/or NB.
Abstract reproduced from the open-access article under the recorded cc by licence; © the authors. PMID 41977352 · DOI · PMC13072898
Key reviews and meta-analyses
The most cited review-type papers among PubMed’s best-match results for this heading, listed with bibliographic details only. Follow the links for the full text and access terms.
- Research article Basket Trials in Oncology: A Trade-Off Between Complexity and Efficiency [5]
- Research article Systematic review and meta-analysis of prevalence of dermatological toxicities associated with vemurafenib treatment in patients with melanoma [6]
- Research article Treating malignant melanoma when a rare BRAF V600M mutation is present: case report and literature review [7]
- Research article Efficacy and toxicity of vemurafenib and cobimetinib in relation to plasma concentrations, after administration via feeding tube in patients with BRAF-mutated thyroid cancer: a case series and review of literature [8]
- Research article Targeted Therapy With Vemurafenib in Brazilian Children With Refractory Langerhans Cell Histiocytosis: Two Case Reports and Review of Literature [9]
- Research article [Erdheim-Chester disease-A histiocytic multisystem disease with unusual renal involvement] [10]
References
- Alabi S, Jaime-Figueroa S, Yao Z, Gao Y, Hines J, Samarasinghe KTG, et al.. Mutant-selective degradation by BRAF-targeting PROTACs. . 2021. PMID 33568647 · DOI · PMC7876048
- Bourland J, Fradette J, Auger FA. Tissue-engineered 3D melanoma model with blood and lymphatic capillaries for drug development. . 2018. PMID 30181613 · DOI · PMC6123405
- Fattore L, Ruggiero CF, Pisanu ME, Liguoro D, Cerri A, Costantini S, et al.. Reprogramming miRNAs global expression orchestrates development of drug resistance in BRAF mutated melanoma. . 2019. PMID 30254376 · DOI · PMC6748102
- Alfei S, Signorello MG, Tirendi S, Khaledizadeh E, Giordani P, Reggio C, et al.. Quaternary Phosphonium Salts Outperformed Vemurafenib (PLX) and Etoposide Against BRAFV600D,V600E PLX-Resistant Melanoma and MDR Neuroblastoma, Exhibiting No/Low Toxicity on 3T3/HaCaT Cells. . 2026. PMID 41977352 · DOI · PMC13072898
- Cunanan KM, Gonen M, Shen R, Hyman DM, Riely GJ, Begg CB, et al.. Basket Trials in Oncology: A Trade-Off Between Complexity and Efficiency. J Clin Oncol. 2017. PMID 27893325 · DOI · PMC5559900
- Chen P, Chen F, Zhou B. Systematic review and meta-analysis of prevalence of dermatological toxicities associated with vemurafenib treatment in patients with melanoma. Clin Exp Dermatol. 2019. PMID 30280426 · DOI
- Popescu A, Haidar A, Anghel RM. Treating malignant melanoma when a rare BRAF V600M mutation is present: case report and literature review. Rom J Intern Med. 2018. PMID 29168975 · DOI
- van Berge Henegouwen JM, van der Wijngaart H, Zeverijn LJ, Hoes LR, Meertens M, Huitema ADR, et al.. Efficacy and toxicity of vemurafenib and cobimetinib in relation to plasma concentrations, after administration via feeding tube in patients with BRAF-mutated thyroid cancer: a case series and review of literature. Cancer Chemother Pharmacol. 2022. PMID 35598186 · DOI · PMC9300506
- Silva KAS, Magalhães IMQS, Benincasa DER, Cecconello DK, Michalowski MB. Targeted Therapy With Vemurafenib in Brazilian Children With Refractory Langerhans Cell Histiocytosis: Two Case Reports and Review of Literature. Cancer Rep (Hoboken). 2024. PMID 39190425 · DOI · PMC11348861
- Dasdelen S, Büschek F. [Erdheim-Chester disease-A histiocytic multisystem disease with unusual renal involvement]. Internist (Berl). 2020. PMID 32785740 · DOI
About this article
- Sources
- NLM Medical Subject Headings (definition, synonyms); NCBI PubMed (counts, identifiers); Europe PMC (open-access abstracts and citation counts).
- Verification
- Every reference was resolved against PubMed on 2026-10-31; title, year and DOI matched.
- Review state
- automated source verified; no human expert review has been recorded yet.
- Version
- 1, last updated 2026-10-31.
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Evidence strength
EvidenceHelix's computed view of the study designs behind this topic: how common meta-analyses, randomized trials and reviews are, how recent the literature is, and a plain-language reading. It describes the design mix of the literature. It is not a quality appraisal of any study, and it is not medical advice.