FDA Fast-Tracks Rare Blood Disease Treatment Zelboraf

Vemurafenib (Zelboraf), a treatment for mutated forms of BRAF V6000 with the rare blood condition Erdheim-Chester disease (ECD), has been granted priority review and a supplemental New Drug Application (sNDA) by the US Food and Drug Administration (FDA).

The inhibitor treatment’s Priority Review designation is supplemented by a Breakthrough Therapy Designation, granted to treatments with potential to alter the course of treatment for conditions, and intended to help expedite the development and review of said treatments towards FDA approval.

This designation is particularly important for the conditions Zelboraf treats. ECD is characterized by its abnormal multiplication of histiocytes, a form of white blood cells. BRAF, a pathway-signaling protein found in cells, aids cell growth and survival control.

Since being co-developed under a 2006 license and agreement between Roche Group and Plexxikon, Zelboraf has served as a prescription medicine for adult patients with melanoma involving abnormal BRAF genes.

The sNDA for Zelboraf included data from the VE-BASKET study, an open-label Phase II trial that enrolled patients with diseases that had underlying genetic profiles matching to medicine’s action and mechanism. This type of study is done to collect data faster and expedite the development of crucial treatments — especially for rare diseases.

The FDA decision regarding Zelboraf’s approval for ECD with BRAF V6000 Mutations expected by December 2017.

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