Zelboraf First Drug Approved for Erdheim-Chester Disease
November 6, 2017 – The U.S. FDA has approved Zelboraf® (vemurafenib), manufactured by Genentech, to treat Erdheim-Chester Disease (ECD) in patients who have the BRAF V600E mutation. This is a new indication for the drug, which was originally approved to treat patients who had inoperable or metastatic melanoma with the BRAF V600E mutation.
ECD is a rare form of slow-growing blood cancer. It is typically found in patients who are in their 50s or 60s, most of them male. Just over half of all ECD patients carry BRAF V600E mutations. Life expectancy following diagnosis is limited. The cancer, which begins in the bone marrow, leads to excess production of histiocytes, a type of white blood cell, and can spread to numerous organs and tissues, including the lungs, heart, and brain.
Zelboraf, a kinase inhibitor, blocks enzymes that contribute to the growth of cancer cells. It was found to reduce tumor sizes in just over half of patients with BRAF-V600-mutation positive ECD in a clinical study. Recommended dosing is 960mg every 12 hours by mouth, with or without food.
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