FDA Approves First Treatment for Rare Blood Cancer

NOVEMBER 06, 2017
Officials with the FDA have extended the approval of vemurafenib (Zelboraf, Genentech) to include the treatment of certain adult patients with Erdheim-Chester Disease (ECD), a rare blood cancer, making it the first approved treatment for ECD. 

Vemurafenib is indicated to treat patients whose cancer cells have a specific genetic mutation known as BRAF V600. It was previously approved in 2011 to treat certain patients with melanoma that have the BRAF V600E mutation. The therapy is a kinase inhibitor that works by blocking certain enzymes that promote cell growth.

ECD originates in the bone marrow and causes an increased production of histiocytes, a type of white blood cell, which can result in tumors infiltrating many organs and tissues throughout the body. Currently, patients with ECD have very limited life expectancies.

The approval is based on data from the phase 2 VE-BASKET clinical trial that included 22 patients with BRAF-V600 mutation positive ECD. The trial evaluated the efficacy of vemurafenib for the treatment of ECD by measuring the percent of patients who experienced a complete or partial reduction in tumor size (overall response rate). Fifty percent of patients experienced a partial response and 4.5% experienced a complete response.

The most common severe adverse reactions reported in the trial included the development of new skin cancers, high blood pressure, rash, and joint paint.

Other common adverse effects include small, raised bumps, hair loss, change in the heart’s electrical activity, and skin growths.


FDA Approves Zelboraf (Vemurafenib) for Erdheim-Chester Disease with BRAF V600 Mutation [news release]. San Francisco. Genentech’s website. . Accessed November 6, 2017

FDA approves first treatment for certain patients with Erdheim-Chester Disease, a rare blood cancer [news release]. FDA’s website. . Accessed November 6, 2017.