The US Food and Drug Administration (FDA) expanded the approval of vemurafenib to include the treatment of BRAF V600 mutation-positive Erdheim-Chester disease (ECD), Roche announced in a news release.

ECD is a rare blood cancer — estimates state fewer than 500 cases in the United States — characterized by slow growth and increased production of histiocytes, a class of white blood cell. Approximately 50% of patients with ECD are BRAF V600 mutation-positive.

The FDA based its approval on results from the phase 2 VE-BASKET study in which patients with BRAF V600 mutation-positive diseases, including ECD, were treated with vemurafenib. The 22 patients with ECD in the study exhibited a best overall response rate of 54.5%, and at the median follow-up time of 26.6 months, the duration of response was not evaluable.

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The most frequently reported grade 3 or greater adverse events (AEs) (10% or more of patients) included new malignancies, hypertension, rash, and joint pain.  The most common AEs (50% or more of patients) included pain, rash, hair loss, fatigue, change in heart rhythm, and skin tags.

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Vemurafenib is the first drug to be approved for the treatment of ECD. Vemurafenib was previously granted Priority Review and Breakthrough Therapy Designation by the FDA for ECD with BRAF V600 mutation.


FDA approves Zelboraf (vemurafenib) for Erdheim-Chester disease with BRAF V600 mutation [press release]. Basel, Switzerland: Roche; November 7, 2017. Accessed November 7, 2017.