Posted by Michael Wonder on 06 Nov 2017
FDA approves first treatment for certain patients with Erdheim-Chester Disease, a rare blood cancer
6 November 2017 - FDA expands approval of Zelboraf (vemurafenib) to treat certain adult patients with Erdheim-Chester Disease, a rare cancer of the blood.
Zelboraf is indicated to treat patients whose cancer cells have a specific genetic mutation known as BRAF V600. This is the first FDA-approved treatment for ECD.
The FDA granted this application priority review and breakthrough therapy designations for this indication. Zelboraf also received orphan drug designation for this indication, which provides incentives to assist and encourage the development of drugs for rare diseases.
