Vemurafenib (Zelboraf)

General information

Class/mechanism: Tyrosine kinase inhibitor of some mutated forms of BRAF serine/threonine kinase, including BRAF V600E. Also has been observed to inhibit other tyrosine kinases such as RAF1, ARAF, wild-type BRAF, SRMS, ACK1, MAP4K5, and FGR. Certain BRAF mutations, such as V600E, result in constitutive activation of cell proliferation pathways and tumor growth.
Route: PO
Extravasation: n/a

For conciseness and simplicity, HemOnc.org currently will focus on treatment regimens and not list information such as: renal/hepatic dose adjustments, metabolism (including CYP450), excretion, monitoring parameters (although this will be considered for checklists), or manufacturer. Instead, for the most current information, please refer to your preferred pharmacopeias such as Micromedex, Lexicomp, UpToDate (courtesy of Lexicomp), or the prescribing information.

Diseases for which it is used

• BRAF-mutated colorectal cancer
• BRAF-mutated melanoma
• BRAF-mutated tumors (tissue-agnostic)
• Erdheim-Chester disease
• Hairy cell leukemia
• Langerhans cell histiocytosis
• Multiple myeloma
• Thyroid cancer

More drug information

• DailyMed information
• Vemurafenib (Zelboraf) patient drug information (UpToDate)

History of changes in FDA indication

• 8/17/2011: Initial FDA approval for the treatment of patients with unresectable or metastatic melanoma with Biomarkers#BRAF V600E mutation. (Based on BRIM2, BRIM-3, and McArthur et al. 2017)
  • Limitation: vemurafenib is not recommended for use in patients with wild-type BRAF melanoma.
• 11/6/2017: Granted regular FDA approval for the treatment of patients with Erdheim-Chester Disease (ECD) with BRAF V600 mutation. (Based on VE-BASKET ECD expansion)

Also known as

• Code names: PLX4032, RG7204, RO5185426
• Brand name: Zelboraf