A New Treatment for a Rare Blood Cancer

Zelboraf (vemurafenib) approved to treat Erdheim-Chester disease

(RxWiki News) The US Food and Drug Administration (FDA) has approved the first treatment for a rare type of blood cancer.

The agency expanded the approval of Zelboraf (vemurafenib) to allow it to be used to treat Erdheim-Chester disease (ECD). This marks the first approved treatment for this rare blood cancer.

Zelboraf was first approved in 2011 to treat melanoma in patients who have a particular gene mutation. Now, the FDA has approved it to treat ECD in patients with a similar gene mutation.

ECD, a blood cancer that starts in the bone marrow, is estimated to affect between 600 and 700 people around the world, according to the FDA. Zelboraf is intended to block enzymes that allow the cancer cells to grow.

The FDA noted that, overall, this medication appeared safe and effective in a trial of 22 patients. Half of the patients saw their tumors shrink, and one patient saw tumors completely disappear.

This medication does pose a risk of some severe side effects. These include the development of new cancers, tumor growth in some patients with melanoma, allergic and skin reactions, liver damage, heart problems, kidney failure, eye reactions, immune reactions and others. This drug can also harm developing fetuses.

More common side effects of Zelboraf included skin growths, changes in the electrical activity of the heart, fatigue, hair loss, rashes and joint pain. Always discuss the side effects of any new medication you're taking with your health care provider.

The FDA granted its approval of Zelboraf to Hoffman-LaRoche, Inc.