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News From the Food and Drug Administration
December 19, 2017

First Treatment Approved for Rare Blood Cancer

JAMA. 2017;318(23):2288. doi:10.1001/jama.2017.18871

By expanding its approval for the kinase inhibitor vemurafenib, the FDA has sanctioned the first treatment in the United States for Erdheim-Chester disease (ECD), a rare blood cancer that originates in bone marrow.

Marketed as Zelboraf, vemurafenib is indicated for adults with ECD who have a specific mutation in the BRAF gene. The drug was first approved in 2011 for patients with melanoma who have the same mutation. The expanded approval “demonstrates how we can apply knowledge of the underlying genetic characteristics of certain malignancies to other cancers,” Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence, said in a statement.