Erdheim-Chester disease (ECD) is a rare type of slow-growing blood cancer caused by overproduction of cells called histiocytes, according to the National Institutes of Health. Histiocytes, a family of white blood cells, normally destroy foreign substances and protect the body from infection. In patients with ECD, the excess production of histiocytes leads to inflammation that can damage organs and tissues throughout the body, causing them to become thickened, dense, and fibrotic.

Symptoms usually appear between the ages of 40 and 60 and can vary widely, including bone pain, hormonal problems from damage to the pituitary gland, headaches, seizures, cognitive impairment, or problems with movement or sensation. People with this condition can also have shortness of breath, heart or kidney disease, protruding eyes, skin growths or infertility, and can experience fever, night sweats, fatigue, weakness and weight loss. While ECD is an extremely rare disease, affected patients have very limited life expectancies.

Source: ECD Global Alliance

Hoffman-La Roche, a subsidiary of Roche Holdings (OTCQX: RHHBY), received FDA approval today for label expansion of Zelboraf (vemurafenib), a kinase inhibitor that blocks certain enzymes that promote cell growth, to include the treatment of certain adult patients with ECD. Zelboraf is indicated to treat patients whose cancer cells have a specific genetic mutation known as BRAF V600, which appears in about 54% of patients with ECD. This is the first ever FDA-approved treatment for ECD.

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. 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.

– 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

Please email us at [email protected] to see our Case Studies and Testimonials.

Please click here for information on our new trading platform.

Please click here to see our weekly newsletter.