The US Food and Drug Administration (FDA) approved vemurafenib, a BRAFV600 inhibitor, for Erdheim-Chester Disease (ECD), according to a press release.1 This is the first-ever drug approval for the blood cancer, which, although rare, carries a poor prognosis.

Approval was based on results from the non-randomized phase 2 VE-BASKET study (ClinicalTrials.gov Identifier: NCT01524978), for which patients with BRAFV600-positive cancers were enrolled and assigned to receive vemurafenib.

Of the 22 patients with BRAFV600-positive ECD, the best overall response rate was 54.5%: 11 patients had a partial response and 1 patient had a complete response. At the median follow-up of 26.6 months, the median duration of response was not estimable.

Severe adverse events noted with vemurafenib include second primary malignancies, anaphylaxis, QT prolongation, and liver damage.

About one-half of patients with ECD have BRAFV600 disease.

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The FDA granted vemurafenib Priority Review as well as Breakthrough Therapy and Orphan Drug statuses. In 2011, vemurafenib was approved to treat BRAFV600-mutated metastatic melanoma.

Reference

  1. FDA approves first treatment for certain patients with Erdheim-Chester Disease, a rare blood cancer [news release]. US Food and Drug Administration; November 6, 2017. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm583931.htm?utm_campaign=11062017_PR_FDA%20approves%20treatment%20Erdheim-Chester&utm_medium=email&utm_source=Eloqua. Accessed November 6, 2016.