FDA approves Zelboraf for rare blood disease

FDA approved an sNDA from the Genentech Inc. unit of Roche (SIX:ROG; OTCQX:RHHBY) for Zelboraf vemurafenib to treat BRAF V600 mutation-positive Erdheim-Chester disease. Genentech said Zelboraf is the first FDA-approved treatment for the rare blood disease characterized by abnormal multiplication of white blood cells called histiocytes. The approval came ahead of the drug's Dec. 7 PDUFA date.

Genentech spokesperson Courtney Siekirk told BioCentury the company believes that Zelboraf is the first targeted therapy approved based on data from a basket study. Zelboraf's approval for the blood disease was based on data from the Phase II VE-BASKET trial in which the drug led to a best overall response rate (ORR) of 54.5% in 22 patients with BRAF V600 mutation-positive Erdheim-Chester disease. ...