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.
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.
- 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.