Therapeutic Response Statement (Sensitivity)

This therapeutic response statement Supports the relationship that BRAF p.V600K status confers therapeutic sensitivity to Vemurafenib in patients with Erdheim-Chester Disease.

The U.S. Food and Drug Administration granted approval to vemurafenib for the treatment of patients with Erdheim-Chester Disease and a BRAF V600 mutation.

This statement is based on a regulatory approval from the Food and Drug Administration:

ZELBORAF is indicated for the treatment of patients with Erdheim-Chester Disease with BRAF V600 mutation.

Citation

Genentech, Inc. Zelboraf (vemurafenib) [package insert]. U.S. Food and Drug Administration website. https://www.accessdata.fda.gov/drugsatfda_docs/label/2020/202429s019lbl.pdf. Revised May 2020. Accessed October 30, 2024. Genentech, Inc. Zelboraf (vemurafenib) [package insert]. U.S. Food and Drug Administration website. https://www.accessdata.fda.gov/drugsatfda_docs/label/2017/202429s016lbl.pdf. Revised November 2017. Accessed October 6, 2026.

Contributions

Date	Contributor	Description
2024-10-30	Van Allen lab	Initial access of FDA approvals
2017-11-06	Food and Drug Administration	Indication received traditional approval.