Zelboraf (vemurafenib) has been approved by the U.S. Food and Drug Administration as the first drug to treat Erdheim-Chester Disease, a rare but deadly blood cancer. The approval covers patients who have a genetic mutation called BRAF V600.
Erdheim-Chester Disease is a slow-growing cancer that originates in bone marrow, causing a spike in a type of white blood cell called a histiocyte. This can spur tumors that develop in the heart, lung, brain and elsewhere, the FDA said Monday in a news release. The cancer only affects about 700 people worldwide, about half of whom have the BRAF V600 mutation.
Life expectancy is short among patients, the agency said.
"This [drug] was first approved in 2011 to treat certain patients with melanoma [skin cancer] that harbor the BRAF V600 mutation, and we are now bringing the therapy to patients with a rare cancer with no approved therapies," said Dr. Richard Pazdur, director of the agency's Oncology Center of Excellence.
Zelboraf is a kinase inhibitor that's designed to inhibit enzymes that spur cancer cell growth. The most common side effects include joint pain, skin rash, hair loss, fatigue and heart problems, the FDA said.
Less common but more severe adverse reactions could include development of other cancers, severe skin reactions, liver damage and kidney failure. Pregnant women shouldn't take the drug, since it could harm a developing fetus, the FDA said.
The drug is produced by the Swiss pharmaceutical firm Hoffman-LaRoche.
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