Vemurafenib, a B-Raf enzyme inhibitor, has been approved by FDA for treatment of rare blood cancer patients i.e. Erdheim-Chester Disease (ECD) having BRAF V600 mutation. This article discusses the detailed prescribing information for Vemurafenib. Erdheim-Chester Disease (ECD) Erdheim-Chester Disease (ECD) is a life-threatening rare disease characterized by excessive multiplication of histiocytes, a type of white blood cells.
Excess histiocytes can result in tumors infiltrating many organs throughout the body, including the heart, lungs, brain and others. ECD is estimated to affect 600-700 patients worldwide and patients generally have very low life expectancy. Approval Of Vemurafenib Vemurafenib is a BRAF enzyme inhibitor initially approved in 2011 for the treatment of late-stage melanoma patients having BRAF V600E mutation. Evidence Approximately 54% ECD patients also have the BRAF…