MedPath

Vemurafenib

Generic Name
Vemurafenib
Brand Names
Zelboraf
Drug Type
Small Molecule
Chemical Formula
C23H18ClF2N3O3S
CAS Number
918504-65-1
Unique Ingredient Identifier
207SMY3FQT
Background

Vemurafenib is a competitive kinase inhibitor with activity against BRAF kinase with mutations like V600E. It exerts its function by binding to the ATP-binding domain of the mutant BRAF. Vemurafenib was co-developed by Roche and Plexxikon and it obtained its FDA approval on August 17, 2011, under the company Hoffmann La Roche. After approval, Roche in collaboration with Genentech launched a broad development program.

Indication

Vemurafenib is approved since 2011 for the treatment of metastatic melanoma with a mutation on BRAF in the valine located in the exon 15 at codon 600, this mutation is denominated as V600E. The V600E mutation, a substitution of glutamic acid for valine, accounts for 54% of the cases of cutaneous melanoma.

Vemurafenib approval was extended in 2017, for its use as a treatment of adult patients with Erdheim-Chester Disease whose cancer cells present BRAF V600 mutation. Erdheim-Chester disease is an extremely rare histiocyte cell disorder that affects large bones, large vessels, central nervous system, as well as, skin and lungs. It is reported an association of Erdheim-Chester disease and V600E mutation.

Associated Conditions
Metastatic Melanoma, Refractory Lung Non-Small Cell Carcinoma, Unresectable Melanoma, Refractory Erdheim-Chester disease
Clinical Trials
Related News

Penn Medicine's Lynn Schuchter: Three Decades of Pioneering Melanoma Treatment and Research

• Dr. Lynn Schuchter has dedicated 30 years at Penn Medicine to advancing melanoma treatment, leading groundbreaking research in targeted therapies and immunotherapy approaches. • As director of the Tara Miller Melanoma Center and former division chief of hematology/oncology, Dr. Schuchter helped expand the faculty from 35 to 150 positions while maintaining focus on patient-centered care. • Under her leadership, significant breakthroughs were achieved in melanoma treatment, including the development of BRAF-targeted therapies and combination treatments that have dramatically improved patient outcomes.

Atezolizumab Switch After Targeted Therapy Shows Survival Trend in BRAF+ Melanoma

• Patients with BRAF V600–positive melanoma showed a trend towards improved overall survival with early switch to atezolizumab after vemurafenib/cobimetinib run-in. • The phase 2 ImmunoCobiVem trial explored the efficacy of switching to atezolizumab versus continuing targeted therapy in this patient population. • Median overall survival was 49.6 months in the atezolizumab arm compared to 40.2 months in the continuous targeted therapy arm. • Progression-free survival was longer with continuous targeted therapy, but subsequent crossover to the alternative treatment showed potential benefits.

European Market Access Hurdles Loom for PD-1/PD-L1 Immunotherapies in Oncology

• Novel immunotherapies targeting PD-1/PD-L1 pathways show promising clinical results in multiple cancers, with potential for durable responses and combination treatments. • European payers face significant budget challenges due to high therapy costs, indefinite dosing schedules, and broad patient populations across multiple tumor types. • Healthcare systems are likely to implement strict access controls including budget caps, payment-by-results schemes, and specialist center restrictions to manage costs.
© Copyright 2025. All Rights Reserved by MedPath
HomeTerms & ConditionsPrivacy Policy