marked.bio

BRAF inhibitor · small molecule

vemurafenib

14 active trials across 11 of our biomarkers, targeting BRAF V600. 7 active approvals for a biomarker on marked.bio.

Also known as PLX4032, RO5185426, Zelboraf.

Counts only trials that select patients by one of the biomarkers on marked.bio, not every trial of this drug.

Approvals and pivotal results

Biomarker-selected approvals in the United States and the European Union, with the pivotal trial's results and side effects where we have them. Results from different trials are not directly comparable.

TherapyBiomarkerTumourUnited StatesEuropean Union
vemurafenib
FDA, 2017
BRAF V600 mutation Label 1.2: ECD with BRAF V600 mutation. No EU equivalent. Year confirmed: FDA approval announced 06/11/2017. Source
ZelborafBroader label 		BRAF V600E Erdheim-Chester disease Approved 2017 Not approved

VE-BASKET (ECD cohort) single-arm, 22 patients
Response 55% (32-76)Lasting not reached

Stopped for side effects 32%

Trial details and side effects
Patients
BRAF V600 Erdheim-Chester disease (NCT01524978)
Most common side effects
arthralgia, rash, alopecia, fatigue, QT prolongation, skin papilloma (each over 50%), cutaneous squamous cell carcinoma 36% (22 patients)
Notes
Zelboraf US label (Trial 4).
Source
US label
atezolizumab with cobimetinib + vemurafenib
FDA, 2020
unresectable or metastatic, BRAF V600 mutation-positive Label 1.4: BRAF V600 mutation-positive. No EU equivalent. Year confirmed: FDA approval announced 30/07/2020. Source
FDA, 2024
unresectable or metastatic, BRAF V600 mutation-positive Subcutaneous atezolizumab approved 12/09/2024 for all adult IV Tecentriq indications, including melanoma; label 1.4. Source
Tecentriq, Tecentriq HybrezaBroader labelatezolizumab pagecobimetinib page 		BRAF V600E melanoma Approved 2020 Not approved

IMspire150 vs placebo + cobimetinib + vemurafenib, 514 randomised
PFS 15.1 months vs 10.6 months, HR 0.78 (0.63-0.97)

Trial details and side effects
Patients
first-line BRAF V600 melanoma (NCT02908672)
Most common side effects
rash, musculoskeletal pain, nausea, fatigue, hepatotoxicity, pyrexia
Notes
Atezolizumab added to cobimetinib and vemurafenib. From the FDA approval notice.
Source
FDA approval summary
cobimetinib with vemurafenib
FDA, 2015
unresectable or metastatic, BRAF V600E or V600K Label 1.1. Year confirmed: FDA approval announced 10/11/2015. Source
EU (EMA), 2015
unresectable or metastatic, BRAF V600 mutation MA 20/11/2015. Source
CotellicBroader labelcobimetinib page 		BRAF V600E melanoma Approved 2015 Approved 2015

In the US label

coBRIM vs placebo + vemurafenib, 247 vs 248 patients
PFS 12.3 months vs 7.2 months, HR 0.56 (0.45-0.70)Response 70% vs 50%OS 22.3 months vs 17.4 months, HR 0.69 (0.54-0.88)

Stopped for side effects 15%

Trial details and side effects
Patients
BRAF V600E or V600K melanoma (NCT01689519)
Most common side effects
diarrhoea 60%, photosensitivity 46%, nausea 41%, pyrexia 28%, vomiting 24% (247 patients)
Notes
Cobimetinib with vemurafenib. Cotellic US label rev. 07/2026 (Trial 1).
Source
US label

In the EU product information

coBRIM vs placebo + vemurafenib, 247 vs 248 patients
PFS 12.3 months vs 7.2 months, HR 0.58 (0.46-0.72)Response 70% vs 50%OS 22.3 months vs 17.4 months, HR 0.70 (0.55-0.90)

Trial details and side effects
Patients
BRAF V600E or V600K melanoma (NCT01689519)
Most common side effects
diarrhoea 60%, photosensitivity 46%, nausea 41%, pyrexia 28%, vomiting 24% (247 patients)
Notes
EU SmPC section 5.1 (coBRIM; PFS January 2015 cut, final OS August 2015). Side effects from the US label.
Source
EU product information
vemurafenib
FDA, 2011
unresectable or metastatic, BRAF V600E Label 1.1: BRAF V600E (not indicated for wild-type). Year confirmed: FDA approval announced 17/08/2011. Source
EU (EMA), 2012
unresectable or metastatic, BRAF V600 mutation-positive MA 17/02/2012. EU label: BRAF V600 mutation-positive. Source
Zelboraf 		BRAF V600E melanoma Approved 2011 Approved 2012

In the US label

BRIM-3 vs dacarbazine, 337 vs 338 patients
PFS 5.3 months vs 1.6 months, HR 0.26 (0.20-0.33)Response 48% vs 5.5%OS 13.6 months vs 10.3 months, HR 0.47 (0.35-0.62)

Stopped for side effects 7%

Trial details and side effects
Patients
first-line BRAF V600E melanoma (NCT01006980)
Most common side effects
arthralgia 53%, alopecia 45%, fatigue 38%, rash 37%, nausea 35%, photosensitivity 33%, cutaneous squamous cell carcinoma 24% (336 patients)
Notes
Zelboraf US label rev. 07/2026 (Trial 1).
Source
US label

In the EU product information

BRIM-3 vs dacarbazine, 337 vs 338 patients
PFS 6.9 months vs 1.6 months, HR 0.38 (0.32-0.46)Response 48% vs 5.5%OS HR 0.78 (0.64-0.94), censored at crossover

Trial details and side effects
Patients
first-line BRAF V600E melanoma (NCT01006980)
Most common side effects
arthralgia 53%, alopecia 45%, fatigue 38%, rash 37%, nausea 35%, photosensitivity 33%, cutaneous squamous cell carcinoma 24% (336 patients)
Notes
EU SmPC section 5.1 (NO25026/BRIM-3, February 2012 PFS cut and December 2012 OS cut). Side effects from the US label.
Source
EU product information

Biomarkers

BiomarkerActive trialsAll trialsFurthest active phase
BRAF V600E 14 111 Phase 3
ALK fusion 2 5 Phase 2
ROS1 fusion 2 2 Phase 2
BRCA1/2 mutation 1 1 Phase 2
EGFR exon 19 deletion / L858R 1 1 Phase 2
KRAS G12C 1 3 Phase 2
NTRK1/2/3 fusion 1 1 Phase 2
RET fusion or mutation 1 3 Phase 2
KRAS G12D 0 2 –
MSI-H / dMMR 0 1 –
PIK3CA activating mutation 0 1 –

Sponsors

Industry sponsors leading active biomarker-selected trials of vemurafenib.

  1. Roche 1
  2. Shanghai Kechow Pharma 1

Trials

14 of 112 trials shown

  1. Efficacy and Safety of Tunlametinib Plus Vemurafenib in Patients With BRAF V600E-mutant Metastatic Colorectal Cancer

    Recruiting Phase 3 Shanghai Kechow Pharma Started 2023Primary completion 2026-12 NCT06008119

    vemurafenib FDA EUbevacizumabcetuximab FDA EU

    Efficacy and Safety of Tunlametinib Plus Vemurafenib in Patients With BRAF V600E-mutant Metastatic Colorectal Cancer

    BRAF V600EColorectal Cancer Metastatic

  2. DETERMINE Trial Treatment Arm 05: Vemurafenib in Combination With Cobimetinib in Adult Patients With BRAF Positive Cancers.

    Recruiting Phase 2/3 Cancer Research UK Started 2023Primary completion 2029-10 NCT05768178

    vemurafenib FDA EUcobimetinib FDA EU

    Confirmed diagnosis of a malignancy harbouring any actionable BRAF V600 mutation using an analytically validated next-generation sequencing method.

    BRAF V600EHaematological Malignancy, Melanoma, Thyroid Cancer, Papillary, Ovarian Neoplasms

  3. Vemurafenib and Cobimetinib for the Treatment of Patients With High Risk Differentiated Thyroid Carcinoma With BRAFV600E Mutation

    Recruiting Phase 2 City of Hope Medical Center Started 2024Primary completion 2026-11 NCT06440850

    cobimetinib FDA EURecombinant Thyrotropin Alfavemurafenib FDA EU

    Known positive BRAFV600E mutation (determined on a previous analysis and/or on a representative formalin-fixed par…

    BRAF V600EThyroid Gland Follicular Carcinoma, Thyroid Gland Oncocytic Carcinoma, Thyroid Gland Papillary Carcinoma

  4. Irinotecan Hydrochloride Liposome Injection (Ⅱ) Combined with Fluorouracil, Folinic Acid, Vermofenib and Cetuximab in First-line Treatment of BRAFV600E Mutated Advanced Colorectal Cancer

    Recruiting Phase 2 Shanghai Changzheng Hospital Started 2024Primary completion 2026-08 NCT06603376

    FOLFIRIvemurafenib FDA EUcetuximab FDA EUbevacizumab

    Patients with BRAFV600E mutation confirmed by tissue or blood testing.

    BRAF V600EStatus may be outdated, last updated 2024-09-19Colorectal Carcinoma

  5. A Study of Vemurafenib and Obinutuzumab Compared to Cladribine and Rituximab in People With Hairy Cell Leukemia (HCL)

    Recruiting Phase 2 Memorial Sloan Kettering Cancer Center Started 2024Primary completion 2027-09 NCT06561360

    vemurafenib FDA EUObinutuzumabcladribinerituximab

    Presence of BRAF V600E mutation as confirmed by PCR, NGS or immunohistochemistry.

    BRAF V600EHairy Cell Leukemia

  6. PLATFORM Study of Precision Medicine for Rare Tumors

    Recruiting Phase 2 Cancer Institute and Hospital, Chinese Academy of Medical Sciences Started 2020Primary completion 2026-07 NCT04423185

    aumolertinibdacomitinib FDA EUalectinibcrizotinibpyrotinibimatinibniraparibpalbociclibvemurafenibsintilimabatezolizumab

    when obtaining EGFR mutation, ALK fusion, ROS-1 fusion, C-MET amplification, C-MET mutation, BRAF mutation, BRCA1/2 mu…

    EGFR exon 19 deletion / L858RBRAF V600EBRCA1/2 mutationALK fusionROS1 fusionRare Tumor

  7. A Study of Multiple Therapies in Biomarker-selected Participants With Resectable Stages IB-III Non-small Cell Lung Cancer (NSCLC)

    Recruiting Phase 2 Roche Started 2020Primary completion 2026-11 NCT04302025

    alectinibentrectinibvemurafenibcobimetinibpralsetinibatezolizumabSBRTChemotherapydivarasib

    …on in ≥ 1% tumor cells as determined by Food and Drug Administration (FDA)-approved test, KRAS G12C mutation

    KRAS G12CBRAF V600EALK fusionROS1 fusionRET fusion or mutationNTRK1/2/3 fusionNon-small Cell Lung Cancer

  8. Combination or Sequence of Vemurafenib, Cobimetinib, and Atezolizumab in High-risk, Resectable Melanoma

    Active, not recruiting Phase 2 Fondazione Melanoma Onlus Started 2020Primary completion 2023-12 NCT04722575

    cobimetinib FDA EUvemurafenib FDA EUatezolizumab FDA

    …urafenib, cobImetinib, and atezolizuMab in Patients With High-risk, Surgically Resectable BRAF Mutated and Wild-type Melanoma"

    BRAF V600EMelanoma

  9. Vemurafenib and Cobimetinib in Treating Patients With BRAF V600E Mutation Positive Craniopharyngioma

    Active, not recruiting Phase 2 Alliance for Clinical Trials in Oncology Started 2018Primary completion 2023-09 NCT03224767

    vemurafenib FDA EUcobimetinib FDA EU

    Vemurafenib and Cobimetinib in Treating Patients With BRAF V600E Mutation Positive Craniopharyngioma

    BRAF V600EBRAF V600E Mutation Present, Papillary Craniopharyngioma

  10. Atezolizumab With Chemotherapy in Treating Patients With Anaplastic or Poorly Differentiated Thyroid Cancer

    Active, not recruiting Phase 2 M.D. Anderson Cancer Center Started 2017Primary completion 2027-07 NCT03181100

    atezolizumab FDAbevacizumabcobimetinib FDA EUnab-paclitaxelpaclitaxelvemurafenib FDA EU

    ADDITIONAL INCLUSION CRITERIA FOR BRAF MUTATION (COHORT 1): Patients with a BRAFV600E mutation being considered for the triplet combination (vemurafenib + cobimetinib + atezol…

    BRAF V600EMetastatic Thyroid Gland Carcinoma, Poorly Differentiated Thyroid Gland Carcinoma, Stage IVA Thyroid Gland Anaplastic Carcinoma AJCC v8, Stage IVB Thyroid Gland Anaplastic Carcinoma AJCC v8

  11. Phase 2 Study Assessing Secured Access to Vemurafenib for Patients With Tumors Harboring BRAF Genomic Alterations

    Active, not recruiting Phase 2 UNICANCER Started 2014Primary completion 2019-05 NCT02304809

    vemurafenib FDA EU

    Patient with BRAF V600 mutation determined by the INCa platforms on the primary and/or metastatic lesion in the …

    BRAF V600ESolid Tumors, Hematologic Cancers, Metastatic Cancers

  12. A Phase I/II Trial of Vemurafenib and Metformin to Melanoma Patients

    Recruiting Phase 1/2 University of Louisville Started 2012Primary completion 2025-06 NCT01638676

    vemurafenib FDA EUmetformin

    Patients with histological confirmed BRAFV600E melanoma (Stage IIIC or Stage IV, American Joint Commission on Cancer);

    BRAF V600EStatus may be outdated, last updated 2021-10-29Melanoma

  13. Vemurafenib Neoadjuvant Trial in Locally Advanced Thyroid Cancer

    Active, not recruiting Phase 2 M.D. Anderson Cancer Center Started 2012Primary completion 2028-09 NCT01709292

    vemurafenib FDA EU

    BRAF V600E mutation detected in the primary tumor or the recurrent/persistent tumor.

    BRAF V600EThyroid Cancer

  14. XL888 + Vemurafenib + Cobimetinib for Unresectable BRAF Mutated Stage III/IV Melanoma

    Active, not recruiting Phase 1 H. Lee Moffitt Cancer Center and Research Institute Started 2016Primary completion 2019-10 NCT02721459

    XL888vemurafenib FDA EUcobimetinib FDA EU

    Must have cytologically or histologically-confirmed unresectable melanoma that harbors a BRAF V600 mutation determined by pyrosequencing assay or equivalent genotyping assay in a Clinical …

    BRAF V600EMelanoma, Skin Cancer