The U.S. Food and Drug Administration Monday granted accelerated approval for Zelboraf (ingredient: vemurafenib) to treat patients with Erdheim-Chester Disease (ECD), a rare cancer of the blood.

ECD is a rare slow-growing blood cancer that originates in the bone marrow and is estimated to affect 600 to 700 patients worldwide. Patients suffering from ECD have limited life expectancies because this disease is characterized by the abnormal multiplication of a specific type of white blood cells called histiocytes.

Excess histiocytes can result in tumors infiltrating many organs and tissues throughout the body, including the heart, lungs, and brain. Approximately 54 percent of patients with ECD have the specific genetic mutation known as BRAF V600.

Zelboraf is a kinase inhibitor that works by blocking certain enzymes that promote cell growth and is the first FDA-approved treatment for ECD. Zelboraf is also specifically targeted for patients with BRAF V600.

The efficacy of Zelboraf was studied in 22 patients with BRAF-V600-mutation-positive ECD. The trial measured the percentage of patients who experienced a complete or partial reduction in tumor size (overall response rate). In the experiment, 11 patients (50 percent) underwent a partial response, and one patient (4.5 percent) experienced a complete response.

Common side effects of Zelboraf include joint pain, small, raised bumps, hair loss, fatigue, change in the heart’s electrical activity, and skin growths.

Severe side effects of Zelboraf include the development of new cancers, severe skin reactions, heart abnormalities, liver damage, severe reactions in the eye, immune reactions after receiving radiation treatment, kidney failure and thickening of tissue in the hands and feet. Pregnant women should be advised of the potential risk to the fetus if considering taking Zelboraf.

“Today’s approval of Zelboraf for patients with ECD demonstrates how we can apply knowledge of the underlying genetic characteristics of certain malignancies to other cancers,” said Richard Pazdur, director of the FDA’s Oncology Center of Excellence. “This product was first approved in 2011 to treat certain patients with melanoma that harbor the BRAF V600E mutation, and we are now bringing the therapy to patients with rare cancer with no approved therapies.”

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