Summaries of Must-Read Clinical Literature, Guidelines, and FDA Actions
Zelboraf Approved for Erdheim-Chester Disease
FDA news release; 2017 Nov 6
The FDA expanded the approval of Zelboraf (vemurafenib) to include the treatment of certain adult patients with Erdheim-Chester Disease (ECD).
Indications: Zelboraf is a kinase inhibitor indicated for the treatment of patients with ECD with BRAF V600 mutation.
Dosage and administration: Recommended dose is 960 mg orally twice daily taken approximately 12 hours apart.
Efficacy and safety: Approval is based on results of a study involving 22 individuals with BRAF-V600-mutation positive ECD. 11 patients experienced a partial response and 1 a complete response.
Side effects/risks: The most common adverse reactions are arthralgia, rash maculo-papular, alopecia, fatigue, electrocardiogram QT interval prolonged, and skin papilloma.
FDA approves first treatment for certain patients with Erdheim-Chester Disease, a rare blood cancer. [news release]. Silver Spring, MD: FDA. 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, 2017.
Zelboraf [package insert]. South San Francisco, CA: Genentech USA, Inc. 2017. https://www.gene.com/download/pdf/zelboraf_prescribing.pdf. Accessed November 6, 2017.