The FDA expanded the indication for vemurafenib (Zelboraf, Genentech) to include the treatment of certain adults with Erdheim-Chester disease (ECD), a rare hematologic malignancy.
The first FDA-approved treatment for ECD, vemurafenib is indicated to treat patients whose cancer cells have the BRAF V600 genetic mutation. ECD is a slow-growing non–Langerhans cell blood cancer that originates in the bone marrow and causes an increased production of histiocytes. Excess histiocytes can