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.
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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
History of changes in FDA indication
- 8/17/2011: Initial FDA approval for the treatment of patients with unresectable or metastatic melanoma with BRAF V600E mutation as detected by an FDA-approved test. (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