Zelboraf (vemurafenib) has been approved for treatment of adult patients with a rare cancer of the blood. It is the first medication approved by the US Food and Drug Administration (FDA) for treatment of Erdheim-Chester Disease (ECD). 

ECD is a rare blood cancer originating in the bone marrow. It is caused by an elevated production of a particular type of white blood cell called a histiocyte. This over-production can result in the formation of tumors in organs and tissues throughout the body. Hoffman-LaRoche’s drug was approved for patients with a specific genetic mutation known as BRAF V600, which is found in more than half of those with ECD. A clinical trial to test the efficacy of Zelboraf studied 22 patients with the BRAF V600 mutation. Of these, eleven patients experienced a partial response to the protein kinase inhibitor, with one patient exhibiting a complete response to the drug. 

Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research, explained this latest approval demonstrates how an understanding of “underlying genetic characteristics” of certain cancers can be used to develop new treatments for other cancers. 

“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 a rare cancer with no approved therapies,” he stated. Zelboraf inhibits cancer growth by blocking certain enzymes that promote cell growth. Then drug is already indicated for a type of skin cancer that cannot be removed using surgery and which has the BRAF gene. 

Zelboraf was granted Priority Review, Breakthrough Therapy, and Orphan Drug designation by the FDA. Orphan Drug status is granted to medications used to treat rare diseases and conditions. In June 2017, the FDA announced plans to eliminate its orphan drug designation backlog and to respond to future applications within 90 days. An orphan drug treats a disease affecting less than 200,000 people in the United States, such as amyotrophic lateral sclerosis, Lennox-Gastaut syndrome and mantle cell lymphoma.  

Common Side Effects of Zelboraf

• Joint pain
• Small raised bumps
• Hair loss
• Fatigue
• Changes in the heart's electrical activity
• Skin growths 

Severe Side Effects

• Development of new cancers
• Tumor growth in patients with BRAF wild-type melanoma
• Hypersensitivity reactions
• Severe skin reactions
• Heart abnormalities
• Liver damage
• Photosensitivity
• Sever reactions in the eye
• Kidney failure
• Immune reactions after receiving radiation treatment
• Thickening of tissue in the hands and feet
• It may also cause harm to a developing fetus

For more information on Zelboraf, visit https://www.roche.com/products/product-details.htm?productId=6bc1de7f-cbc6-4e30-ba69-fee04fb053b8