FDA approves first treatment for Erdheim-Chester disease

The US Food and Drug Administration yesterday expanded the approval of Zelboraf (vemurafenib) to include the treatment of certain adult patients with Erdheim-Chester disease (ECD), a rare cancer of the blood.

Zelboraf, which is marketed by Swiss pharma giant Roche (ROG: SIX), 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. The approval for vemurafenib came about one ahead of a deadline set by the Prescription Drug User Fee Act (PDUFA).