FDA approves Zelboraf for Erdheim-Chester disease.- Genentech.

The FDA has approved Zelboraf (vemurafenib), from Genentech, for Erdheim-Chester disease (ECD) with BRAF V600 mutation.

The approval is based on data from the Phase II VE-BASKET study. Basket studies use an innovative clinical trial design that helps collect data faster and may accelerate the development of medicines for diseases with high unmet need. Instead of enrolling people based primarily on their disease or its location, basket studies match a disease’s underlying genetic profile to the mechanism of action of the medicine. Final results of VE-BASKET for the 22 people with ECD showed a best overall response rate of 54.5 percent. The most common Grade 3 or higher adverse events were new skin cancers, high blood pressure, rash and joint pain. The most common adverse events were joint pain, rash, hair loss, fatigue, change in heart rhythm and skin tags.

Comment: Zelboraf monotherapy was approved for the treatment of people with unresectable or metastatic melanoma with BRAF V600E mutation in 2011. The FDA previously granted Priority Review and Breakthrough Therapy Designation to Zelboraf for ECD with BRAF V600 mutation.